Nexviadyme, (avalglucosidase alfa), Sanofi’s next-generation enzyme replacement therapy for Pompe disease, will now be available in the U.K.
Articles by Margarida Maia, PhD
FORTIS, a Phase 1/2 clinical trial of an experimental gene therapy called AT845, is due to restart in adults…
The U.S. Food and Drug Administration (FDA) has delayed a decision on whether to approve cipaglucosidase alfa, the biologic component…
A number of enzymes involved in synthesizing glycogen – the molecule that accumulates to toxic levels in Pompe disease…
A toddler girl with infantile-onset Pompe disease is able to walk independently and lives with a healthy heart after…
Age and the use of an assistive device like a walker may tell how well a patient with late-onset…
Pain is relevant but appears to be unrelated to damage to small nerve fibers in the skin of late-onset…
The same common GAA gene variants that cause Pompe disease can lead to symptoms first appearing at very different ages,…
Adults with late-onset Pompe disease (LOPD) report worse physical health and quality of life than their counterparts with other…
A better understanding of Pompe disease-causing genetic mutations may help with early treatment intervention, even if symptoms are subtle,…