Advocacy intelligence hub — real-time data for patient organizations
University of Missouri-Columbia — NA
Denali Therapeutics Inc. — PHASE1
AskBio Inc — PHASE1, PHASE2
Shionogi — PHASE2
Genzyme, a Sanofi Company — PHASE4
Oculox Technologies SA — NA
Erasmus Medical Center — PHASE4
GeneCradle Inc — PHASE1, PHASE2
Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
FINANCIAL LANDSCAPE SUMMARY
Total programs
Open now
Copay cards
Copay Assistance2
Pombiliti and Opfolda
Amicus Therapeutics, Inc.
Myozyme
Genzyme Corporation
Pombiliti and Opfolda
(cipaglucosidase alfa-atga and miglustat)Orphan drugAmicus Therapeutics, Inc.
Nexviazyme
(AVALGLUCOSIDASE ALFA-NGPT)Orphan drugGenzyme Corporation
Myozyme
(Recombinant human acid alpha-glucosidase; alglucosidase alfa)Orphan drugGenzyme Corporation
Omeprazole
(OMEPRAZOLE)Orphan drugstandardPD-Rx Pharmaceuticals, Inc.
Proton Pump Inhibitor [EPC]
12.1 Mechanism of Action Omeprazole belongs to a class of antisecretory compounds, the substituted benzimidazoles, that suppress gastric acid secretio...
Pompe Disease Newborn Screening Shows 60% Faster Treatment
States implementing Pompe newborn screening reported 60% reduction in time to treatment initiation.
Newborn Screening Expands to Include More Rare Diseases
The Advisory Committee recommended adding Duchenne MD and Pompe disease to the Recommended Uniform Screening Panel.
Browse all Glycogen storage disease due to acid maltase deficiency news →
Tahseen Mozaffar, MD, MD
University of California Irvine Health
📍 ORANGE, CA
Ozlem Goker-Alpan, MD
Lysosomal & Rare Disorders Research & Treatment Center, Inc.
📍 Fairfax, Virginia
Joseph Rossano, MD, M.D
Children's Hospital of Philadelphia
📍 PHILADELPHIA, PA
Whu-Liang Hwu, MD, PhD
National Taiwan University Hospital
Filip K Knop, MD, PhD
University Hospital, Gentofte, Copenhagen
Use Central Contact
Astellas Pharma Inc
View all Glycogen storage disease due to acid maltase deficiency specialists →