Pompe disease and treatments highlighted in Trump's address - ABC News
Treatment for Adult-onset Pompe Disease Tried in First U.S. Patient | Dana Foundation
Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease | Neurology
Diagnosis and treatment of late-onset Pompe disease in the Middle East and North Africa region: consensus recommendations from an expert group | BMC Neurology | Full Text
Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational study | Orphanet Journal of Rare Diseases | Full Text
Pompe Disease » Powell Center for Rare Disease Research and Therapy » College of Medicine » University of Florida
Lysosomal Storage Disease | Division of Medical Genetics | Stanford Medicine
Clinical manifestation of late onset Pompe disease patients in Hong Kong - Neuromuscular Disorders
Muscle MRI Findings in Childhood/Adult Onset Pompe Disease Correlate with Muscle Function | PLOS ONE
Long-term effects of enzyme replacement therapy in an elderly cohort of late -onset Pompe disease - Neuromuscular Disorders
Uživatel DailyRounds | Marrow na Twitteru: „The @US_FDA Approved Nexviazyme (avalglucosidase alfa-ngpt) For Late-Onset Pompe Disease Recommended dose: 20 mg/kg for patients ≥30 kg OR 40 mg/kg for patients <30 kg | #
Pompes disease
What Are the Symptoms of Pompe Disease? | Pompe Disease News