MaineHealth Barbara Bush Children's Hospital is crucial to so many families, some of which are facing rare and life-altering ...

Pompe disease treatment market grows with advances in ERT and gene therapy, improving patient outcomes and access.

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.

Sanofi has added another string to its Pompe disease therapy bow ... the market four years later to treat late-onset Pompe in children aged over eight. In 2010, Lumizyme's label was expanded ...