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Community DC Susan Hedstrom Foundation for Prader-WIlli Research · Community D.C.

0:00
Speaker 1: Good morning, and welcome to another edition of Community DC.
0:03
Speaker 1: I'm your host Dennis Glasgow. This morning, we welcome to
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Speaker 1: the program for the first time Susan Headstrom. She is
0:08
Speaker 1: the executive director for the Foundation for the Prodder Willie Research,
0:12
Speaker 1: also known as FPWR. It's a nonprofit organization dedicated to
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Speaker 1: eliminating the challenges of PWS. Their primary goal is to
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Speaker 1: advance research to develop effective treatments and an eventual cure.
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Speaker 1: And for quick context, PWS is a complex, rare genetic
0:30
Speaker 1: disorder caused by an abnormality on the fifteenth chromosome. It
0:34
Speaker 1: affects approximately one in fifteen thousand births and impacts development, metabolism,
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Speaker 1: and behavior. And will also be visiting with Kristin White.
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Speaker 1: She is local and on the board at FPWR, and
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Speaker 1: one of her children has PWS, and with her husband,
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Speaker 1: has spearheaded funding and education, among many other things. A
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Speaker 1: story that she'll share with us. Here's my conversation with
0:55
Speaker 1: both Susan and Kristen. I hope you enjoy it as
0:57
Speaker 1: much as I did. Good morning, Susan, Good morning. It
0:59
Speaker 1: is a place to have you on the program here
1:01
Speaker 1: at Community DC. I know you're based in San Diego.
1:03
Speaker 1: But we're going to have an opportunity to talk to
1:05
Speaker 1: somebody locally that's been dealing with with PWR, and her
1:09
Speaker 1: name is Kristin and it's an extraordinary story and she's
1:12
Speaker 1: also on your board too. I'd like to do this though.
1:14
Speaker 1: I did an intro and I tried to explain PWR,
1:17
Speaker 1: but I know I didn't do a justice. And I
1:20
Speaker 1: know you've been with the organization since twenty thirteen as
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Speaker 1: executive director and you have a good hold on what
1:24
Speaker 1: you do. Can you tell us exactly what it is?
1:27
Speaker 2: Sure?
1:28
Speaker 3: Well, I'd be remissed if I didn't start by saying
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Speaker 3: that fpw.
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Speaker 2: BAR was created by parents.
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Speaker 3: Like so many of us in the rare disease space,
1:36
Speaker 3: we find ourselves in this position and in the case
1:39
Speaker 3: of Product Willy syndrome, research was significantly lacking. So we
1:44
Speaker 3: had these go get them parents who really saw a
1:46
Speaker 3: need for research and improving the lives of people with
1:49
Speaker 3: proder Willi syndrome. So they established the foundation for Product
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Speaker 3: Willy Syndrome with one singular focus mission to eliminate the
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Speaker 3: challenges through research therapeutic development. So what does that mean?
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Speaker 3: We fund heading edge science, We importantly connect research around
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Speaker 3: the world, because collaboration and research is so incredibly important
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Speaker 3: if you want to find answers. We maintain the largest
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Speaker 3: patient registry for people with PWS, and of course we
2:20
Speaker 3: work to ensure families have access to the latest information
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Speaker 3: and resources because knowledge, if it's not in the hands
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Speaker 3: of the people who need it, is not going to
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Speaker 3: help them make better outcomes.
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Speaker 1: All right, So the organization goes all the way back
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Speaker 1: to two thousand and three and it was parent led
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Speaker 1: as you had mentioned, if you could break down about PWR,
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Speaker 1: about what actually the syndrome is and what I'm sure
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Speaker 1: that every child is a little bit different, but what
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Speaker 1: exactly is it that you can describe in people so
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Speaker 1: they can try to relate about what different parents and
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Speaker 1: children are going through right now?
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Speaker 3: Sure, so, proder Willy syndrome we call it PWS for short.
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Speaker 3: It's incredibly rare. Those people have never heard of it.
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Speaker 3: It affects about one in fifteen thousand births, so not
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Speaker 3: a whole lot of people with PWS. But ironically, the
3:08
Speaker 3: more you're in the community, the more you start to
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Speaker 3: meet others who have heard of someone or know somebody
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Speaker 3: who has this syndrome. The hallmark symptom is a word
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Speaker 3: that most people have never heard. It's hyperphasia. Hyperphasia is
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Speaker 3: this intense, persistent hunger. Essentially, your brain does not receive
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Speaker 3: the signals it needs to tell you that you're full,
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Speaker 3: so even after eating a full meal, you still feel hungry.
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Speaker 3: Oftentimes people will tell me, well, I always feel hungry,
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Speaker 3: maybe I have PWS.
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Speaker 2: This is an insatiable hunger.
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Speaker 3: People with PWS will go to great links to obtain food.
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Speaker 3: They will eat food off the floor, they will go
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Speaker 3: through trash cans, they will steal food because they are
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Speaker 3: ravenously hungry. So this, you know, this drive to eat,
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Speaker 3: It can be overwhelming and it truly does dominate their
4:05
Speaker 3: lives as well as the lives of the family and
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Speaker 3: the caretakers. Not to diminish, there are many other symptoms
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Speaker 3: as well. Hyperphasia, of course, is the hallmark symptom, but
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Speaker 3: people with PWIS they also have developmental delays, so oftentimes
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Speaker 3: they'll be in special education programs or day programs as adults.
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Speaker 3: They have hormonal challenges, they have behavioral difficulties which are
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Speaker 3: very much life limitters, as well as other health concerns.
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Speaker 3: It is truly a complex disorder and it requires lifelong management.
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Speaker 1: Susan. When it comes to diagnosis and all the research
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Speaker 1: that's happening right now, we'll talk about data and all
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Speaker 1: the other things that are going on, but just in
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Speaker 1: general terms, is it more predisposed to boys and girls?
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Speaker 1: And how soon can somebody be diagnosed? Just in general.
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Speaker 3: Terms, so it affects all ages and races equally engenders,
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Speaker 3: so it does not discriminate. It is a genetic anomaly
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Speaker 3: that happens across the board. As far as diagnosis goes,
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Speaker 3: most individuals will be diagnosed within a few weeks at
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Speaker 3: the hospital. Oftentimes, newborn babies will have failure to thrive,
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Speaker 3: so they cannot drink.
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Speaker 2: They kept nurse or drink from a bottle.
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Speaker 3: They have to have an ENNG two placed through their nose,
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Speaker 3: and eventually some of them will go to have a
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Speaker 3: G two, which is a placement in their stomach for feeding.
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Speaker 3: They are also floppy like rag dolls. So when you
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Speaker 3: see this combination of failure to thrive with the floppiness,
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Speaker 3: many doctors have been trained to test for produ really syndrome. However,
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Speaker 3: this doesn't always happen and oftentimes families will be sent
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Speaker 3: home without a diagnosis, where they begin the rare journey
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Speaker 3: of trying to find that diagnosis and it can take
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Speaker 3: years to find one if it's not done, you know,
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Speaker 3: in the first few months of life.
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Speaker 1: All when it comes to PWS, I know that you're
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Speaker 1: giving some data the one in fifteen thousand, But do
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Speaker 1: you know approximately the latest data you have of how
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Speaker 1: many children do you have it right now? In the
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Speaker 1: United States?
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Speaker 3: That is a really hard number for us to calculate
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Speaker 3: because not all individuals identify within our organizations. However, we
6:17
Speaker 3: did recently do a data pool of insurance claims and
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Speaker 3: we found that about fourteen thousand people with PWS are
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Speaker 3: here in the United States.
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Speaker 1: All right, So you mentioned there's no curative correct.
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Speaker 2: Currently, there is no cure for prider release INNDOME.
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Speaker 3: So most of our interventions are environmental.
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Speaker 1: Okay, very good. So with that said, when it comes
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Speaker 1: to daily life and what parents can be up against
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Speaker 1: and siblings and everything else, and we're talking in generalities again,
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Speaker 1: what's day to day like for families and the children
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Speaker 1: that have the PWS.
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Speaker 3: Sure, well, PWS it impacts every decision a family makes
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Speaker 3: every day. Most families they have to create a highly
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Speaker 3: structured environment around food. So imagine a house. The pantries
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Speaker 3: and refrigerators are locked, meals are going to be planned,
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Speaker 3: they're going to be served on a schedule, and food
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Speaker 3: can't simply be left out on a counter. So when
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Speaker 3: you walk into the home of a person with product
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Speaker 3: really syndrome, you will not see food anywhere.
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Speaker 2: For many people, this sounds unusual. I could say.
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Speaker 3: People will often come to my home and they find
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Speaker 3: it strange that they think we're just very, very clean.
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Speaker 2: There's no food to be seen.
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Speaker 3: But this is how we have to live in order
7:33
Speaker 3: to keep our loved ones safe. Because they can't just
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Speaker 3: be disciplined or have willpower to avoid taking food. We
7:41
Speaker 3: have to physically secure that food. We call it food
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Speaker 3: security in our community. Behavior and mental health, in addition
7:49
Speaker 3: to the food challenges, are also life limiting for people
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Speaker 3: with PWS and their family. So people with PWS, they
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Speaker 3: often have significant behavior outbursts as adults.
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Speaker 2: This can be particularly difficult.
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Speaker 3: To imagine, especially when you're out in the community. So
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Speaker 3: imagine having a full grown adult. You know your full
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Speaker 3: grown son or daughter. You're going to the grocery store
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Speaker 3: and they have a toddler age tantrum, yelling, screaming, throwing.
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Speaker 2: Something, or maybe this is at an airport.
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Speaker 3: This isn't only socially unacceptable, it can actually be unsafe
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Speaker 3: for the person with PWS. So these things all have
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Speaker 3: to be kept in mind as we're planning out our day.
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Speaker 3: Can our loved one make it to the grocery store?
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Speaker 3: Do I need to leave them at home? If I
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Speaker 3: need to leave them at home, who will supervise them
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Speaker 3: while I'm out? All of these things have to be
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Speaker 3: balanced with life.
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Speaker 1: Let's talk a little bit about funding. How are you
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Speaker 1: currently funded?
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Speaker 3: FPWR is almost entirely funded by community fundraisers, So parents
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Speaker 3: like myself who are who's fundraisers in their community to
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Speaker 3: raise funds for research.
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Speaker 1: Now there is a big donate button and also join
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Speaker 1: us in the website. Well, let's get the website right now, everybody,
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Speaker 1: and we'll give it at the end again.
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Speaker 2: Sure.
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Speaker 3: The website is www dot FPWR dot org.
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Speaker 1: One of the things I found, Susan, and you've been
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Speaker 1: with the organization a while. When I talk to nonprofits,
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Speaker 1: especially if there's a disease that is in running rampant
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Speaker 1: and millions of people have it, funding is always difficult.
9:27
Speaker 1: The funding is difficult for everybody. Sometimes it's usually a
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Speaker 1: small organization, it's a two or three person team, and
9:33
Speaker 1: we get all that. Can you talk a little bit,
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Speaker 1: Susan about this disease and the acknowledgment about how many
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Speaker 1: people have it or don't have it, and how funning
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Speaker 1: and you advocating for what you need to do to
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Speaker 1: get the funds in how hard it is at times.
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Speaker 3: You know, funding for rare disease is certainly it's a
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Speaker 3: rare fundraising conundrum because you cannot rely necessarily on the
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Speaker 3: random act of kindness from strangers. It really comes from
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Speaker 3: people who know somebody with proder really syndrome and are
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Speaker 3: moved by that person.
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Speaker 2: People with PWS are lovable.
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Speaker 3: They are so incredibly bright and social and kind, and
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Speaker 3: when you meet a person with PWS, it's really hard
10:19
Speaker 3: to not just fall in love with them. So WED,
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Speaker 3: you know, our funding model really is from people who
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Speaker 3: have been moved and loved these individuals with proder really syndrome.
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Speaker 3: So we host small community events throughout the country.
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Speaker 2: We do it's called a one Small Step Walk, which.
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Speaker 3: Is one of my most favorite fundraising events that we
10:42
Speaker 3: host because it's the community like Kristin White, who you've
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Speaker 3: had on your show, hosting an event and inviting all
10:50
Speaker 3: the other families in the area to come out. We
10:53
Speaker 3: set up fundraising pages, we set a goal, but the
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Speaker 3: big part of the program is everyone coming together and
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Speaker 3: getting to see each other and walking in a safe
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Speaker 3: environment with their loved ones and really celebrating the progress
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Speaker 3: that they're making.
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Speaker 1: Outstanding and the join our events is also part of
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Speaker 1: the different things you can check on the website. And
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Speaker 1: a great segue to talk about Kristin. She's been on
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Speaker 1: your board since twenty twenty two. Her son Raphael has
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Speaker 1: the syndrome and she's going to discuss with us. I
11:21
Speaker 1: think it's extraordinary and I'm going to talk to her
11:23
Speaker 1: about this that. You know, it's one thing to deal
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Speaker 1: with the PWS, but then you decide to give even
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Speaker 1: more raised money be on your board and I think
11:31
Speaker 1: that's extraordinary. Can you tell us a little bit about
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Speaker 1: her before we chat with her.
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Speaker 2: Kristin's an amazing go get her.
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Speaker 3: She's been on our board for four years, but she's
11:41
Speaker 3: been actively plugged into our community early on. Many families
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Speaker 3: like Kristin, when they're given the diagnosis.
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Speaker 2: They feel like.
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Speaker 3: Their power has been taken away from them, that there's
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Speaker 3: nothing they can do. They're given this laundry list of
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Speaker 3: all the bad things that are going to come their way.
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Speaker 2: You feel lost.
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Speaker 3: But people like Kristin they take that and they spin
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Speaker 3: it and they say, what can I do to make
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Speaker 3: this better? This future is not acceptable for my loved one.
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Speaker 3: I don't want them to be at home and unable
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Speaker 3: to live independently for the rest of their lives. So
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Speaker 3: I'm going to do something about it. And really that
12:25
Speaker 3: comes down to so much of our organization as parents
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Speaker 3: who say I'm going to make a difference anyway I can.
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Speaker 3: And for many of us, we're not scientists. We don't
12:39
Speaker 3: have personal million dollar checks from out the Gates family.
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Speaker 2: But we can.
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Speaker 3: Bring people together, we can tell our stories, we can
12:47
Speaker 3: talk about pws, and we can raise funds because it
12:50
Speaker 3: is on us to make sure that this research happens
12:53
Speaker 3: so that we can find treatments for our loved ones.
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Speaker 3: Living hungry your entire life is not acceptable, agreed.
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Speaker 2: It shouldn't be this way, and research is going to
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Speaker 2: change that.
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Speaker 1: And with that, here's my conversation with Kristin. Good Morning, Kristin.
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Speaker 2: Good morning, Dennis.
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Speaker 1: Well, we've had an opportunity to talk to Susan so far,
13:14
Speaker 1: and we're going to revisit more of our conversation, but
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Speaker 1: I would love to talk to you about your journey
13:19
Speaker 1: when it comes to FPWR your family. I know you're
13:21
Speaker 1: on the board, you've been on the board for quite
13:23
Speaker 1: some time, but tell us how you got involved.
13:26
Speaker 2: Sure.
13:27
Speaker 4: Well, I'm the proud mom of three kids. I live
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Speaker 4: here in DC and Capitol Hill with my husband and
13:34
Speaker 4: my three sons, and my second son was born in
13:37
Speaker 4: twenty sixteen at Georgetown University Hospital with proter Williams syndrome,
13:43
Speaker 4: which you won't be surprised to hear we had never
13:48
Speaker 4: heard of before, and so you know, really at that moment,
13:52
Speaker 4: our lives changed. And I'll never forget that one of
13:56
Speaker 4: the first calls we got in the hospital was from
13:58
Speaker 4: somebody from FPWR saying, here's some information. You might not
14:02
Speaker 4: have had this specific information before, but we're here to
14:06
Speaker 4: help you, and it made a big difference in the
14:09
Speaker 4: journey that we've been on for the last almost ten
14:11
Speaker 4: years with our son.
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Speaker 1: Well, tell us about the discovery about you know, how
14:15
Speaker 1: you found out and then when you started to educated
14:19
Speaker 1: what you were up against.
14:21
Speaker 4: Sure, well, you know, most families that that I've met
14:28
Speaker 4: in this in this journey have a very similar story
14:31
Speaker 4: of pregnancy. Oftentimes you know, a very uneventful pregnancy, and
14:38
Speaker 4: then you know after their child is born, being in
14:44
Speaker 4: the hospital and having a lot of unanswered questions, which
14:48
Speaker 4: has often meant long nick U stays and and many
14:53
Speaker 4: many tests and and lots of worries.
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Speaker 1: And so.
15:00
Speaker 4: You know, we were relatively lucky in that Raphael received
15:04
Speaker 4: his diagnosis about two weeks after he was born, and
15:09
Speaker 4: so from that moment on we were able to start
15:13
Speaker 4: getting him the best support available to him and have
15:18
Speaker 4: done that ever since. But it's not a because it's
15:21
Speaker 4: not a very common syndrome. It doesn't show up on
15:25
Speaker 4: prenatal testing or newborn screens, and so you're often learning
15:28
Speaker 4: about it after the fact through a series of tests
15:32
Speaker 4: that could also turn up very scary things. And so
15:34
Speaker 4: it's really it's a really tough time for families before
15:37
Speaker 4: they receive their diagnosis.
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Speaker 1: So what is day to day like for him and
15:41
Speaker 1: also the family?
15:44
Speaker 4: Well, Rafa is now nine, almost ten, and he is
15:49
Speaker 4: an incredibly successful second grader. He has a lot of loves.
15:57
Speaker 4: His longtime, longtime love is Power Rangers. He's more recently
16:02
Speaker 4: discovered dance and is in local dance productions and does
16:08
Speaker 4: very well. But you know, our life is not without
16:12
Speaker 4: its challenges. PWS has, you know, serious behavioral issues that
16:18
Speaker 4: accompany this unrelenting hunger, and these kids suffer from a
16:24
Speaker 4: lot of anxiety, and that impacts the whole family. And
16:27
Speaker 4: so we live a life that includes all of that
16:31
Speaker 4: as well as all of the good things.
16:33
Speaker 1: Right and listen, I have a family too, and a
16:36
Speaker 1: daughter is twenty four, and you know there's always chaos
16:39
Speaker 1: in a family. I imagine the one thing beside the
16:42
Speaker 1: education and learning how to deal with everything in the
16:45
Speaker 1: syndrome that the family had to and I'm talking about
16:48
Speaker 1: all the kids in the family, had to adopt a
16:49
Speaker 1: lot of patients.
16:52
Speaker 2: Yes, we've had to be patient.
16:54
Speaker 4: Something I've learned in this journey with PWS is that
16:58
Speaker 4: Rafa can and will learn anything, but it will be
17:02
Speaker 4: on his own time. And so you know that has
17:07
Speaker 4: just meant milestones at different at different times than his brothers.
17:11
Speaker 4: That has sometimes meant our family moving at a different pace.
17:16
Speaker 4: But you know, it's hard to say who's who's harder
17:21
Speaker 4: to teach patients to siblings or parents, But we're all
17:25
Speaker 4: doing our best.
17:26
Speaker 1: Well, I want to talk about you joining the board
17:29
Speaker 1: in twenty twenty two. You know, I think it's one
17:31
Speaker 1: thing to have something happen to the family and then
17:34
Speaker 1: deal with that and all the challenges, But you decided
17:36
Speaker 1: to go to the next level and be a part
17:39
Speaker 1: of the board. Tell us about that decision and why
17:41
Speaker 1: you did it.
17:43
Speaker 4: Sure well, I think you know, one of the greatest
17:46
Speaker 4: things about our community is the resources that we have
17:51
Speaker 4: available to us as families, and FPWR is a big
17:55
Speaker 4: part of that. You know. I was invited to the
17:58
Speaker 4: first conference, you know, shortly after my son was born.
18:03
Speaker 4: I wasn't able to attend, but red materials, stayed up
18:07
Speaker 4: late reading at night, and those were things that gave
18:10
Speaker 4: me hope in those early days. It was really a
18:12
Speaker 4: meaningful part of understanding the diagnosis and trying to get
18:16
Speaker 4: my head around what my son's life would be like.
18:19
Speaker 4: And if you talk to families with kids who have PWS,
18:24
Speaker 4: I think we all believe that there's a better future
18:26
Speaker 4: for our kids because of research and science, and f
18:30
Speaker 4: PWR is driving that research. They are racking up successes
18:36
Speaker 4: and it's played a really important part in how we
18:40
Speaker 4: view the future for our family and for my son,
18:43
Speaker 4: and so had been involved with the foundation through walks
18:48
Speaker 4: and attending conferences where a very small community, we all
18:51
Speaker 4: get to know each other pretty quickly, and was very
18:56
Speaker 4: excited about the opportunity to join the board and be
19:00
Speaker 4: more involved with this organization. And so over the past
19:04
Speaker 4: few years have spent time getting to know the leadership
19:09
Speaker 4: of FPWR and working closely with them, and we're doing
19:12
Speaker 4: our best to sort of support them in this massive
19:14
Speaker 4: mission that they've undertaken.
19:16
Speaker 1: Right, and I also know that fundraising has been just
19:19
Speaker 1: a big deal for you too as as part of
19:20
Speaker 1: being a board member.
19:22
Speaker 2: That's right.
19:23
Speaker 4: Well, as much as I never would have thought that
19:27
Speaker 4: fundraising would be in my on my list of things
19:32
Speaker 4: that I do on a on a yearly basis, we've
19:36
Speaker 4: learned that money buy science and science saves lives, and
19:42
Speaker 4: so in addition to raising awareness, we've put a lot
19:46
Speaker 4: of effort into raising money to support FPWR, And so
19:50
Speaker 4: my husband and I have held an event now here
19:55
Speaker 4: in DC for a couple of years. It's an annual
19:58
Speaker 4: event called Bourbon on the Hill, a rare night for RAFA,
20:02
Speaker 4: and that's our way of bringing our community together to
20:07
Speaker 4: raise funds for research that's important to our family, but
20:11
Speaker 4: also to celebrate and celebrate our successes as well.
20:16
Speaker 1: Well. Ruff sounds like such a nice young man. And
20:19
Speaker 1: I know that a lot has been thrown at you
20:21
Speaker 1: in the family, but I think it's extraordinary or you,
20:23
Speaker 1: your husband and the family are doing so continue success
20:27
Speaker 1: with you being on the board when it comes to
20:29
Speaker 1: PWR and all that you're doing. And I really appreciate
20:31
Speaker 1: you joining us on Community DC. Thank you so much.
20:34
Speaker 2: Thanks Dennis Susan.
20:35
Speaker 1: That was a really nice chat with Kristin, and I
20:38
Speaker 1: learned a lot in the short time that we had
20:40
Speaker 1: with her. You know, I said this when I was
20:43
Speaker 1: talking to her, and I'll say to you again, I
20:45
Speaker 1: think it's extraordinary her parents go through something like this
20:48
Speaker 1: and then decide to be on a board. It's the
20:51
Speaker 1: next level. But the one other thing my takeaway with
20:54
Speaker 1: Kristin being on your board and who has a family
20:56
Speaker 1: member who has PWS, is that she gets to relate
20:59
Speaker 1: to all the other parents that are going through this.
21:01
Speaker 1: And you and I know we want simple acknowledgment in
21:04
Speaker 1: life and we also want to be able to relate
21:06
Speaker 1: to people, and I imagine her as a board member
21:08
Speaker 1: and being relate with other parents who am I going
21:10
Speaker 1: forward through the first time with PWS. It's got to
21:13
Speaker 1: be extraordinary that she can talk about what you've been
21:16
Speaker 1: through for her family. Would I be correct?
21:18
Speaker 2: Absolutely? Our community members.
21:21
Speaker 3: Are all there for the newly diagnosed because we've been there,
21:25
Speaker 3: we've felt it, we know what people are going.
21:28
Speaker 2: Through, and we understand the journey and it's.
21:31
Speaker 3: Truly as unique and it's nice to have someone who's
21:34
Speaker 3: been there just a little bit before you to help
21:37
Speaker 3: kind of guide you down that path.
21:39
Speaker 1: We had mentioned that you've been with the organization for
21:41
Speaker 1: quite some time. From where you started to where you
21:44
Speaker 1: are now and your advocation, how we doing on research?
21:48
Speaker 1: Once again, I know it's hard to raise funds. I
21:50
Speaker 1: know research is probably difficult when you have a rare disease.
21:53
Speaker 1: But from where you started to where you are now,
21:56
Speaker 1: have you made some inroads?
21:58
Speaker 2: Absolutely so.
21:59
Speaker 3: When I I first became involved with this organization, this
22:02
Speaker 3: was over a decade ago, there were no treatments for PWS.
22:07
Speaker 2: There's growth hormone. Growth hormone helps with.
22:11
Speaker 3: Obviously growth, you know, stature but also helps with body composition.
22:14
Speaker 3: But there was nothing for the symptoms that challenge us
22:18
Speaker 3: most nothing for hyperphasia, nothing for the behavior, the mental health.
22:23
Speaker 3: But today we have half a dozen clinical trials ongoing
22:27
Speaker 3: and we have our first FDA improved treatment for hyperphasia.
22:32
Speaker 3: There's a lot of movement that we still need to make.
22:35
Speaker 3: Not every drug is going to work for every individual
22:38
Speaker 3: with PWS. We've seen that, and we still don't have
22:43
Speaker 3: a cure for hyperphasia. So while we are certainly addressing
22:46
Speaker 3: it and treating it and improving upon the symptom, we
22:50
Speaker 3: have a lot of work to go until every person
22:53
Speaker 3: can live at full and independent life.
22:55
Speaker 1: I don't know if you get an opportunity to talk
22:57
Speaker 1: to the doctors and the researchers, but if you do,
23:00
Speaker 1: or your people do, I imagine once again, with funds
23:04
Speaker 1: being tough at times, so it gets a little frustrating
23:06
Speaker 1: because you need millions and millions of dollars for research,
23:08
Speaker 1: especially rare disease. But as you talk to these people
23:11
Speaker 1: in research, how do they feel about the future of it?
23:16
Speaker 2: So we are in communication with a lot of them.
23:19
Speaker 3: I think right now there's a lot of concern about
23:23
Speaker 3: the future of research funding has gotten very, very challenging.
23:27
Speaker 3: Our foundation has seen a threefold increase in applications for.
23:32
Speaker 2: Funding for grants.
23:35
Speaker 3: Unfortunately, we can't fund every grant of application that comes
23:38
Speaker 3: into us, even if they are scientifically emeritis simply our
23:42
Speaker 3: budgets don't they're constrained. What we're seeing is, you know,
23:47
Speaker 3: larger funding sources are not as available as previously, so
23:51
Speaker 3: people are seeking out funding from us. So that's that's
23:55
Speaker 3: been challenging to not be able to address all of
23:58
Speaker 3: the funding needs. And also out of the younger investigators
24:03
Speaker 3: are being pushed out of the labs. They're simply not
24:05
Speaker 3: room for them. So we're concerned that five ten to
24:08
Speaker 3: fifteen years and from now there's going to be a
24:10
Speaker 3: big gap in knowledgeable researchers because our youngest are being
24:17
Speaker 3: forced out of the field.
24:19
Speaker 1: Yeah, it's challenging out there right now, and I know
24:21
Speaker 1: it's a tough landscape for all nonprofits out there, especially yours.
24:25
Speaker 1: With that said, i'd love to ask you because I
24:28
Speaker 1: once again being with the organization, I'm sure it's a
24:31
Speaker 1: struggle and it's tough to hear all the hard stories
24:34
Speaker 1: that parents and families and caregivers are going through. But
24:37
Speaker 1: also I know that there are some special things that
24:39
Speaker 1: happen when you talk to families or when you see
24:41
Speaker 1: something really cool happen. And this is one of my
24:44
Speaker 1: favorite questions of the community c show that we do
24:48
Speaker 1: is maybe asking you a story about this is why
24:50
Speaker 1: we get up every day because something special happened over
24:53
Speaker 1: the last couple of years. Is there something really remarkable
24:56
Speaker 1: or miraculous that you saw with a family that was
24:59
Speaker 1: really cool up and you say, you know, this is cool,
25:02
Speaker 1: this is why we get up every day. Can you
25:04
Speaker 1: share something with us?
25:06
Speaker 2: Oh? Goodness, I mean but immediately comes to mind.
25:12
Speaker 3: The big win for our community was simply getting our
25:15
Speaker 3: first FDA treatment. Because it's not one family, it's hundreds
25:20
Speaker 3: of family who made something like that happen. It starts with,
25:25
Speaker 3: you know, the individuals who take a risk and participate
25:29
Speaker 3: in a clinical trial.
25:30
Speaker 2: We try to make clinical.
25:31
Speaker 3: Trials as safe as we can, but you know, there's
25:35
Speaker 3: always there's oftentimes fear of participating. But hundreds of individuals
25:40
Speaker 3: raised their hands and participated in clinical in the clinical
25:44
Speaker 3: trial for this one drug and many many others, So
25:48
Speaker 3: hats off.
25:49
Speaker 2: To all of them.
25:49
Speaker 3: We've also had thousands of parents who've enrolled in our
25:53
Speaker 3: Global PWS Registry, which is an incredibly important research tool
25:57
Speaker 3: that is showing us trends and helping us improve care
26:01
Speaker 3: for people with PWS. That registry was also a We
26:05
Speaker 3: were able to use data from that registry to support
26:08
Speaker 3: the approval of our first FDA approved treatment for PWS.
26:13
Speaker 3: So all of these patients or parents who are participating
26:17
Speaker 3: in that way are making a huge tremendous difference as well.
26:20
Speaker 3: And then of course the fund raising aspect, so to
26:23
Speaker 3: come up with one story that it's really challenging to
26:28
Speaker 3: do that, but to give praise to all the people
26:31
Speaker 3: who are getting up every day p wos is tough,
26:35
Speaker 3: and to ask them to do one more thing is
26:38
Speaker 3: it's asking them to do a really big lift, and
26:41
Speaker 3: they are because they know how important it is for
26:44
Speaker 3: us to move research forward and find treatments.
26:47
Speaker 2: We've seen that treatments are possible.
26:50
Speaker 3: We need, you know, we need technology and research to
26:53
Speaker 3: advance a little bit.
26:54
Speaker 2: More, but that more treatments are going to be coming.
26:56
Speaker 1: Well, I appreciate you sharing that, and I think that
26:58
Speaker 1: goes back to what you and I were talking about.
27:00
Speaker 1: It about the simple relating that families can do that. I'm
27:04
Speaker 1: not the only one I've shared with my audience that
27:06
Speaker 1: I've had rheumatarta arthritis since I was twenty one. I
27:09
Speaker 1: had this in the late eighties, no Internet, no social media,
27:12
Speaker 1: bad drugs, and when you have something, you think you're
27:15
Speaker 1: the only person on the planet that's experiencing it. It's
27:17
Speaker 1: very isolating. And I can imagine relating to these families
27:20
Speaker 1: and they hear about a disease that they've never heard before.
27:22
Speaker 1: Ever it's now in their family, and they feel like
27:25
Speaker 1: they're on an island by themselves. Here's where you get
27:28
Speaker 1: injected with the other parents, the board members, and the
27:31
Speaker 1: relating that you're not alone. And that's why I think
27:34
Speaker 1: it's cool that you guys do exist absolutely.
27:38
Speaker 3: And you know, when I first received the diagnosis for
27:41
Speaker 3: my own son, it felt very alone. I knew nobody
27:44
Speaker 3: who had a child with product Really syndrom or any
27:47
Speaker 3: rare disease for that matter. I felt no one could
27:50
Speaker 3: possibly understand when.
27:51
Speaker 2: I was going through.
27:53
Speaker 3: And then I was really fortunate that I attended a
27:56
Speaker 3: conference hosted by the Foundation for Product Really Research. That
27:59
Speaker 3: was as my introduction to the foundation, and I looked
28:03
Speaker 3: around a room. I was surrounded not only by parents
28:06
Speaker 3: who had children with PWS, but the researchers whose hearts
28:11
Speaker 3: and minds were tied to this disorder as well, and
28:13
Speaker 3: I realized I'm not alone. There are people who are
28:18
Speaker 3: working together to make the world better for my son
28:22
Speaker 3: and everyone with PWS. And having that moment was incredibly
28:26
Speaker 3: important because no one likes to feel alone.
28:29
Speaker 1: Yeah, I'm glad you mentioned that, because I've experienced that
28:32
Speaker 1: you have and most people have in daily life. Whatever
28:35
Speaker 1: is going on, and there's a hardship, and it is remarkable, folks,
28:39
Speaker 1: to just be able to relate that you are not alone.
28:41
Speaker 1: So those words are very powerful. Susan, we have just
28:44
Speaker 1: about a minute left. I'd like to get some final
28:46
Speaker 1: thoughts from you. Give the website, and let's tell everybody
28:49
Speaker 1: once again how they can be a part of it
28:51
Speaker 1: with donating and joining you. The floor is yours.
28:54
Speaker 2: Oh thank you so much, Dennis so Crowder.
28:57
Speaker 3: Really, syndrome might be rare, but the challenges are credibly profound.
29:01
Speaker 3: The encouraging news, of course, is that research is working.
29:04
Speaker 3: Every discovery is bringing us closer to treatments.
29:08
Speaker 2: One day.
29:08
Speaker 3: I know that people with pws will be able to
29:10
Speaker 3: live healthier and more independent lives. That's why FPDWR exists,
29:15
Speaker 3: That's why we're committed to this work. We would love
29:18
Speaker 3: for people to come and learn more about produd really
29:20
Speaker 3: syndrome on our website FPWR dot org and today tend
29:24
Speaker 3: any one of our fundraising events if they're.
29:26
Speaker 1: So inclined, outstanding, and let's give the website just one
29:30
Speaker 1: more time. And if you're on social media platforms too,
29:32
Speaker 1: whatever people can reach out to you. How do they
29:34
Speaker 1: do that?
29:35
Speaker 2: So we do have.
29:36
Speaker 3: Social media accounts on both Facebook, Instagram and LinkedIn. We
29:39
Speaker 3: would of course love to have more followers. We shared
29:42
Speaker 3: lots of great information about prouder really on those sites,
29:45
Speaker 3: and again people can come and learn more and get
29:47
Speaker 3: involved at FPWR dot org.
29:49
Speaker 1: Outstanding Susan, thanks for making the introduction of Christen so
29:52
Speaker 1: we can meet here in the DC area and all
29:54
Speaker 1: the best to you and your family. Thank you so
29:55
Speaker 1: much for joining us on Community DC. We really appreciate it.
29:58
Speaker 2: Thank you so much. Dennis