About Pompe Disease - United Pompe Foundation
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
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
Newcastle Hospitals Research on X: "The @jwmdrc team has dosed the first patient in Europe as part of a clinical trial to find treatment for late onset pompe disease. A fantastic step
Muscular Dystrophy Association Celebrates FDA Approval of Nexviazyme for Treatment of Pompe disease | Muscular Dystrophy Association
ZiP 006: “Overview of gene therapy for Pompe disease” | Pompe Support Network
Understanding the Global Pompe Disease Market: Diagnosis, Treatment, and Research
Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease | EMBO Molecular Medicine
Is the 6MWT still the best endpoint for Pompe disease clinical trials?
Duke To Begin Clinical Trials For Pompe Disease Gene Therapy This Fall | Duke Health
Qualitative interviews to improve patient-reported outcome measures in late-onset Pompe disease: the patient perspective | Orphanet Journal of Rare Diseases | Full Text
Timeline showing the different clinical trials published since 2001.... | Download Scientific Diagram
Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease - ScienceDirect
Pompe disease diagnosis and management guideline | Genetics in Medicine
Consensus treatment recommendations for late-onset Pompe disease. - Abstract - Europe PMC
Timeline of phase I/II clinical trial for Pompe disease. Following... | Download Scientific Diagram
National Institute of Arthritis and Musculoskeletal and Skin Diseases - Research in a mouse model of Pompe disease at @DukeUniv, funded by @ncats.nih.gov, is now ready for clinical trials in humans. NIAMS
AT845 for Pompe Disease | Pompe Disease News
Pompe disease: pathogenesis, molecular genetics and diagnosis | Aging
Biomolecules | Free Full-Text | Pompe Disease: New Developments in an Old Lysosomal Storage Disorder
Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction: Molecular Therapy - Methods & Clinical Development
