Test Your Knowledge About the Etiology of Pompe Disease
Pompe disease, also known as glycogen storage disease type 2, is an autosomal recessive disorder. Take our quick quiz on its etiology.
POMPE Podcasts
Test Your Knowledge of the Complications of Pompe Disease
Pompe disease varies with the age of onset and rate of disease progression. Take our quick quiz on the complications of this disease.
An Interview With Muscular Dystrophy Association President Donald S. Wood
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Donald S. Wood, PhD, president and CEO of the Muscular Dystrophy Association (MDA).
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…
AATD Podcasts, ALGS Podcasts, GIST Podcasts, LAL-D Podcasts, LCFAOD Podcasts, MTC Podcasts, PAH Podcasts, Podcasts, POMPE Podcasts, SMA Podcasts
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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