Aro Biotherapeutics has dosed the first patient in a Phase Ib trial of ABX1100, a novel treatment for late-onset Pompe disease (LOPD), a rare inherited disorder that causes progressive muscle ...

Robert Califf, MD, former commissioner of the FDA, delivered a keynote address at the 2025 Muscular Dystrophy Association ...

It is a detailed view of the brain like never seen before. In a significant technological leap, University of Florida researchers have created a powerful new computational and artificial intelligence ...

Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its latest therapy for the rare, inherited disorder. The US regulator has approved ...

Megan’s affliction, Pompe disease, is exceedingly rare. The course of the disease varies by patient, but it causes children’s muscles to weaken and, eventually, their lungs may fail.

currently in the phase 1/2 FORTIS study in late-onset Pompe disease (LOPD) in adults. Xork is designed as a pre-treatment to overcome one of the key limitations to adeno-associated virus (AAV ...

Amicus, which focuses on rare diseases, has two medicines in the market: Galafold, a treatment for Fabry disease, and Pombiliti+Opfolda, a combination therapy for Pompe disease. Fabry disease is ...

Analysts raised concerns about competitive pressures in the Pompe disease market, particularly from existing therapies. FX volatility remains a potential risk, with a 1% US dollar fluctuation ...