Amicus Therapeutics (Nasdaq: FOLD), today announced the presentation of new data from clinical and real-world studies of Galafold® (migalastat) in Fabry disease and POMBILITI® + OPFOLDA® ...

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 ...

Maher Tarabishi was the primary caretaker of his son, Wael Tarabishi, 30, who died on Friday, Jan. 23, from complications related to Pompe disease ...

(NEW YORK) — President Donald Trump’s first address to a joint session of Congress happened to fall on Rare Disease Day — a fact he highlighted by recognizing one of his invited guests, Megan Crowley, ...

An Amicus Therapeutics combination treatment for the rare enzyme deficiency Pompe disease now has FDA approval, giving the drugmaker the opportunity to help patients who don’t respond to Sanofi ...

Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.

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 ...

DURHAM, N.C. (WTVD) -- A local family is going to incredible lengths to help a Durham girl battling a rare and fatal illness. Maddison Castillo, 5, was born with Pompe disease, a genetic disorder that ...

Hunter syndrome (MPS II) presentations will include analysis from continued follow-up of Phase 1/2 data for tividenofusp alfa (DNL310), currently under FDA Priority ReviewPreliminary data from Phase 1 ...