Amicus Therapeutics anticipates completing a rolling application by mid-year to seek approval of its investigational therapy AT-GAA for…
Articles by Marta Figueiredo, PhD
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of…
Newly developed nerve cells derived from patients could be used as a model to study neurological involvement in Pompe disease…
Patient-reported outcomes — particularly those related to general physical and arm function — effectively reflect disease severity in people with…
The first wave of COVID-19Â in Europe severely disrupted access to care and raised stress and anxiety in people with…
Bayer reached an agreement to acquire Asklepios BioPharmaceutical (AskBio), including its clinical program of ACT-101, an experimental…
The European Medicines Agency agreed to begin reviewing Sanofi Genzyme’s application requesting the approval of avalglucosidase alfa, its investigational…
Adding a preventive short-course immunomodulatory regimen — rituximab, methotrexate, and intravenous immunoglobulins (IVIG) — to enzyme replacement…
A moss-derived type of enzyme replacement therapy (ERT) for Pompe disease may be more effective than the currently approved…
Sanofi Genzyme’s next-generation enzyme replacement therapy (ERT), avalglucosidase alfa, safely halts disease progression in people with…