Pompe disease is a rare genetic disorder that disables the heart and skeletal muscles. The inherited disorder can develop at any age, although the often-fatal disorder has faster progression and ...

Purpose: Electrocardiogram (ECG) abnormalities are universal in infantile Pompe disease or glycogen storage disease type II, a fatal genetic muscle disorder caused by deficiency of acid α-glucosidase ...

Pompe disease is a rare genetic disorder that impacts both children and adults. It’s important to understand the next steps following a Pompe disease diagnosis to help prepare you and your loved ones ...

Sanofi has two therapies for the rare disorder Pompe disease, one of which is a relatively recent entry into the market. But a drug candidate from Maze Therapeutics would offer advantages over both ...

Slated for an FDA decision last October, Amicus Therapeutics’ Pompe disease bid was foiled by COVID-related travel restrictions. Nearly a year later, the Philadelphia company has gained its ...

CARMEL, Ind. — Every Thursday, 1-year-old Grant and his parents come to IU Health North Hospital so he can receive a five-hour enzyme replacement infusion. It’s something they’ve been doing since he ...

When two of John Crowley’s children were diagnosed with the rare and debilitating Pompe disease, he founded a company that helped develop a successful treatment. Now, he’s running another biotech — ...

Opfolda (miglustat) is a medicine taken with another medicine for certain people with late-onset Pompe disease. Pompe disease is a rare genetic disorder that is caused by an abnormal gene for a ...

Rare genetic disorder affects 1 in 40,000 people. — -- President Donald Trump's first address to a joint session of Congress happened to fall on Rare Disease Day -- a fact he highlighted by ...