Notas del 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.
Palabras clave
rare diseaseDuchennegene therapyfshdFDAVinay Prasad