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Expert Interview

Slingshot members are talking to an expert! The topic is:

Discussing the FDA Approval of Amicus Therapeutics' Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) combo therapy for treating Pompe Disease.

Ticker(s): FOLD

Who's the expert?

Institution: McMaster University

  • Professor of Pediatrics and Medicine, Director of Neuromuscular and Neurometabolic Clinic at McMaster University Medical Center.
  • Manages 21 patients with Pompe Disease and served as a PI for the PROPELL study
  • Research focuses on mitochondrial diseases, muscle disorders, and other neurometabolic disorders, and has been involved in clinical trials in in Duchenne muscular dystrophy, Friedreich's ataxia and Pompe disease

Interview Goal
This conversation will focus on discussing the FDA Approval of Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) combo therapyin patients with late-stage Pompe disease who are not improving on their current enzyme replacement therapy.

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Reason

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