Episode 3: Facioscapulohumeral muscular dystrophy, Duchenne, and the Sarepta-FDA shakeup

Immature Brains: A Child Neurology Podcast di Ali Christy and Sam Mackenzie

Note sull'episodio

In our neuromuscular episode, Sam and Ali interview Kristin Zwickau about the challenges facing pediatric patients with facioscapulohumeral muscular dystrophy and other rare diseases when it comes to drug development. Ali discusses the historical contributions of Charles Bell, William Gowers, and Guillaume Duchenne. Sam closes with a rundown on recent news surrounding Sarepta Therapeutics and the FDA.

Parole chiave
rare diseaseDuchennegene therapyfshdFDAVinay Prasad