درباره این اپیزود
When he founded Cure Rare Disease in 2018, Rich Horgan was primarily focused on finding a treatment for his younger brother Terry, who had an ultra-rare form of Duchenne Muscular Dystrophy (DMD).
Suffering from a disease that has no cure, Terry served as a “selfless, brave, and courageous” figure that Rich continues to draw inspiration from when seeking new, innovative solutions to treat this progressive, neurodegenerative condition.
Join us for this interview as Rich discusses the origins of Cure Rare Disease, Terry’s ongoing legacy, the collaborative effort it takes to advance the drug development journey, the important role of the patient voice when developing treatments, and the future of rare disease research.
Show Notes
- What We Owe Terry Horgan: Reflections from Providers, Family, and Scientists
- Letter From Terry's Parents
- The Disruptors - Meet Rich
- In Vitro Assays for Duchenne Muscular Dystrophy
- 2022 World Congress Video
- Duchenne Muscular Dystrophy Studies
- Rare Disease Research for Drug Development
- Scientific Collaboration Leads to Faster Drug Development
- Cure Rare Disease
انگلیسی
ایالات متحده آمریکا
در این اپیزود
رونوشت 🔗
Are you the producer of this podcast?
Add a podcast transcript
Need Audio-to-Text?
Transcribe with Listen411 in Just 60 Seconds
جستجوی اپیزودهای گذشته
اپیزودهای قبلی Vital Science را جستجو کن.
قسمت های دیگر در این پادکست
Imagine a world where your child has a rare disease that’s so rare, it doesn’t have a name and only a handful of people around the world have a similar condition. It can leave you feeling helpless and alone knowing the odds are stacked against you.
This is the situation Bo Bigelow experienced wi…
The effects of Angelman Syndrome (AS) on young children are debilitating. From an early age, they face an uphill battle with this neurogenetic disease’s unrelenting symptoms, including seizures, cognitive impairments, and loss of motor function.
It can also be tough on caretakers – just ask Dr. A…
Spinocerebellar ataxia type 3 (SCA3) is a neuromuscular disease affecting just 50,000 people in the United States, including three generations of the Klassen family.
Greg Klassen, his father, and his son have all been diagnosed with this rare condition that impacts the central nervous system, a…
For decades, researchers have utilized animal models to help predict the effects of a drug compound in humans.
With recent advances in technology and the passage of the FDA Modernization Act 2.0, researchers are increasingly looking at new methods to refine, reduce, and replace in vivo testing …
18-year-old Emily Whitehead aspires to be a writer, but nothing could have prepared her for the plot twist that struck her as a child.
In the space of just 24 hours, five-year-old Emily suffered from various symptoms that culminated in a diagnosis of acute lymphoblastic leukemia (ALL). After al…
سلب مسئولیت: پادکست و آثار هنری تعبیه شده در این صفحه متعلق به Charles River است که متعلق به صاحب آن است و به Listen Notes، Inc وابسته یا تایید نشده است.
ویرایش
از کمک شما برای بروز نگهداشتن پایگاهدادههای پادکست سپاسگزاریم
