Acid Maltase Deficiency (AMD)

Acid maltase deficiency is also called Pompe disease, glycogenosis type 2, acid-alpha glucosidase deficiency, and lysosomal storage disease. It is a metabolic muscle disorder that interferes with how the body processes food to produce energy.

Featured Article

The Case for Newborn Screening

By Chris Anselmo | July 15, 2019
Featured Article

Ongoing Clinical Trials

By Ori Rokach | July 15, 2019
Picture of two hands using a screwdriver to disassemble a strand of DNA
Featured Article

These 7 Things Can Help You Understand Gene Targeting Therapy

By Amy Madsen | July 15, 2019
Blog Post

FDA Approves PTC Therapeutics’ Emflaza for the Treatment of DMD in Patients Between 2 and 5 Years Old

By Jeanene Swanson | June 10, 2019
Blog Post

Congress is Considering a Law to Strengthen Newborn Screening: Help MDA Promote its Passage

By Brittany Hernandez | June 4, 2019
Blog Post

JAMA Neurology Publishes Article on Newborn Screening and Neuromuscular Disorders

By MDA Staff | May 24, 2019
Featured Article

The Independent Mindset

By Barbara and Jim Twardowski, RN | March 25, 2019
Blog Post

FDA Grants Breakthrough Therapy Designation to Amicus’ Experimental Therapy, AT-GAA, for Late-Onset Pompe Disease

By Jeanene Swanson | March 7, 2019
Blog Post

Clinical Trial Alert: Amicus Therapeutics Seeks Participants for a Phase 3 Late-Onset Pompe Disease Study

By MDA Staff | March 5, 2019
Blog Post

Newborn Screening for DMD and BMD: MDA Needs Your Help

By MDA Staff | January 15, 2019