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 ...

Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe disease.

AUSTIN, Texas — With four children and 10 grandchildren, Austin resident Kelly Kelley has everything to fight for. "They have all come to accept that I'm sick but I'm not done,” Kelley said. "My first ...