Targeting the Toughest Diseases

Targeting Duchenne Muscular Dystrophy


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John Killian’s son was diagnosed with Duchenne muscular dystrophy (DMD) as a toddler. Until recently, children with DMD were not expected to live beyond their teens. The disease weakens the body's muscles and cognitive functions, which can lead to premature death. John’s son is now college-bound, thanks to the help of medical advancements that target the symptoms of the disease. Geneticist Dr. Alison McVie-Wylie and the research team at Vertex hope to move past only treating the symptoms of DMD by addressing the disease at its root cause.

Produced by Bloomberg Media Studios and Vertex Pharmaceuticals.

 

Featured guests:

John Killian is the proud father of Sam Killian, who lives with DMD.

Mena Scavina is a Neurologist and Clinical Director at Nemours Children's Health and a Clinical Care Team Advisor at Parent Project Muscular Dystrophy.

Jon Rey-Hastie is the Co-Founder of Pathfinders Neuromuscular Alliance

Dr. David Altshuler is the Executive Vice President, Global Research and Chief Scientific Officer at Vertex Pharmaceuticals.

 Alison McVie-Wylie is a Vice President leading the Duchenne muscular dystrophy research team at Vertex Pharmaceuticals.

Read more about Vertex's approach to targeting Duchenne muscular dystrophy.
 

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Targeting the Toughest DiseasesBy Vertex Pharmaceuticals

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