Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial - The Lancet Neurology
Marrow on X: "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 | #medtwitter https://t.co/CZN4NK0X2n" /
Pompe disease diagnosis and management guideline | Genetics in Medicine
Managing Late-onset Pompe Disease in the Era of Newborn Screening: Considerations for Disease Monitoring and ERT Initiation | Catalyst Medical Education
Childhood Pompe disease: clinical spectrum and genotype in 31 patients | Orphanet Journal of Rare Diseases | Full Text
Pompe Disease - WikiMSK
Pompe Disease - Symptoms, Causes, Treatment | NORD
Follow‐up of late‐onset Pompe disease patients with muscle magnetic resonance imaging reveals increase in fat replacement in skeletal muscles - Nuñez‐Peralta - 2020 - Journal of Cachexia, Sarcopenia and Muscle - Wiley
Late Onset Pompe Disease Mimicking Rigid Spine Syndrome | Canadian Journal of Neurological Sciences | Cambridge Core