Amicus Therapeutics settled litigation with Teva, preventing a generic version of Galafold for Fabry Disease until 2037, ...

European regulators have for the fourth time issued a negative opinion on PTC's Translarna. Elsewhere, Amylyx read out data ...

Amicus Therapeutics’ first oral drug for the rare condition Fabry disease has been approved by the FDA, giving patients an alternative to conventional injected enzyme replacement therapy.

Amicus also has a second FDA-approved drug in its commercial portfolio. In September 2023, the FDA approved Pombiliti (cipaglucosidase alfa) + Opfolda (miglustat) 65mg capsules, a two-component ...

Deb Jenssen never wanted her children to suffer from the disease that killed her brother at 28. The illness, Duchenne muscular dystrophy, initially manifests in childhood as trouble with strength ...

C, emphasizes the need for newborn screening to detect infantile-onset lysosomal storage diseases and swift treatment.

“This important designation highlights the need for new gene therapies like AMT-191 for patients with Fabry disease with the potential of delivering meaningful benefit given the suboptimal ...

LEXINGTON, Mass. and AMSTERDAM, Oct. 15, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing ...

Allschwil, Switzerland – October 16, 2024 Idorsia Ltd (SIX: IDIA) today announced that new data on aprocitentan, Idorsia’s ...

Oct. 10, 2024 — Inflammation of the arteries is a primary precursor and driver of cardiovascular disease -- the No. 1 killer of people in the United States. This inflammation is associated with ...

Of the hundreds of genetic diseases linked to the X chromosome, Duchenne is among the more common, along with certain forms of hemophilia. Other so-called X-linked disorders include Fabry disease ...