Press Release: Sanofi’s Nexviazyme met all primary and secondary endpoints in infantile-onset Pompe disease phase 3 study

Sanofi’s Nexviazyme met all primary and secondary endpoints in infantile-onset Pompe disease phase 3 studyNexviazyme met its primary endpoint, ...
WebMD

Diagnosing Pompe Disease

Getting a diagnosis of Pompe disease can be tricky. That's partly because the disorder is so rare. It affects only about 1 in every 40,000 people in the United States. “It’s really not on most ...
Healthline

Pompe Disease and Nutrition: A Starter Guide

Pompe disease is a rare metabolic disorder. It’s marked by a deficiency in lysosomal alpha-glucosidase. Your body needs this enzyme to break down stored glucose, which is called glycogen. When your ...
Nasdaq

Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study of AT-GAA at the 24th International Annual Congress of the World Muscle Society

CRANBURY, N.J. and COPENHAGEN, Denmark, Oct. 02, 2019 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced additional positive results from a global Phase 1/2 clinical study ...
Business Wire

Aro Biotherapeutics Receives FDA Rare Pediatric Drug Designation for ABX1100 for the Treatment of Pompe Disease

PHILADELPHIA--(BUSINESS WIRE)--Aro Biotherapeutics, a biotechnology company pioneering the development of tissue-targeted genetic medicines, today announced that the U.S. Food and Drug Administration ...