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AT845 Gene Therapy - AAV8 delivery of the human GAA gene to treat Pompe Disease (Image from Audentes Therapeutics)

August 6, 2020

AT845: Audentes Therapeutics Begins Recruiting Patients for new Phase 1/2 LOPD Gene Therapy Clinical Trial for Pompe Disease (FORTIS)

July 29, 2020

Higher dosing of alglucosidase alfa (ERT) improves outcomes in children with Pompe disease: a clinical study and review of the literature

Pompe Exercise Study

July 29, 2020

Exercise training alone or in combination with high-protein diet in patients with late onset Pompe disease: results of a cross over study

Actus-101 Pompe Disease Gene Therapy Preliminary Results

July 29, 2020

Actus-101 Gene Therapy Promising Early Results (Phase 1/2 study of AAV2/8-LSPhGAA in Late-Onset Pompe Disease)

Higher ERT Dose IOPD

July 22, 2020

Effects of higher and more frequent dosing of alglucosidase alfa (40mg/kg/week ERT dose) and immunomodulation on long‐term clinical outcome of classic infantile Pompe patients

Pompe Disease Respiratory Function

July 19, 2020

Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start

Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease

July 19, 2020

Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease