Recent Posts

• AT845: Audentes Therapeutics Begins Recruiting Patients for new Phase 1/2 LOPD Gene Therapy Clinical Trial for Pompe Disease (FORTIS)
• Higher dosing of alglucosidase alfa (ERT) improves outcomes in children with Pompe disease: a clinical study and review of the literature
• 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 Gene Therapy Promising Early Results (Phase 1/2 study of AAV2/8-LSPhGAA in Late-Onset Pompe Disease)
• 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