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 disease, also known as glycogen storage disease type 2, is an autosomal recessive disorder. Take our quick quiz on its etiology.
Pompe disease varies with the age of onset and rate of disease progression. Take our quick quiz on the complications of this disease.
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Donald S. Wood, PhD, president and CEO of the Muscular Dystrophy Association (MDA).
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…
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,…