POMPE Podcasts

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Dr. Barry Byrne on Recent Therapeutic Developments in Pompe Disease

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Barry Byrne, MD, director of the University of Florida’s Powell Gene Therapy Center in Gainesville. Dr. Byrne has spent years researching potential gene therapies for Pompe disease, comments on the FDA’s recent approval of avalglucosidase alfa-ngpt (Nexviazyme) to treat Pompe disease. Read the full article: Scientists…

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An Interview With Dr. Edward Neilan, Chief Medical Officer at NORD

Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Edward Neilan, MD, chief medical officer at the National Organization for Rare Disorders (NORD). This is a new position at NORD, which serves as the umbrella organization for some 330 disease-specific patient advocacy groups. NORD also sponsors the annual Rare Diseases and Orphan Products Breakthrough Summit,…

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