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

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 ...

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

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

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.

“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 ...

Pompe Disease is a metabolic disorder that causes a buildup of glycogen in the body's cells, damaging cells and impairing the function of organs and tissues, especially muscles. Fabry Disease is a ...

First patient dosed with ISP-001 shows reduced urinary GAG level, functional improvements surpassing enzyme replacement ...

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 ...

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