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.

From her perch on the edge of a bathtub, red-haired Denise Delfau swung her pretty legs and contentedly scribbled in a notebook. It was all quite jolly, except for the water that occasionally splashed ...

Purpose: A clinical trial was conducted to evaluate the safety and efficacy of alglucosidase alfa in infants and children with advanced Pompe disease. Methods: Open-label, multicenter study of IV ...

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 ...

Not just my usual body ache and pain caused by the symptoms of living with Pompe disease, but every breath I take feels like it is a real effort and struggle.