Air Force veteran Melissa Hay battled years of misdiagnosis before discovering she had Fabry disease—a rare, life-threatening ...

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.

"We are committed to delivering innovative therapies and solutions for people living with Fabry disease, their families and caregivers." A biopharmaceutical company, Protalix leverages its plant ...

Background: Neuropathic pain is one of the key features of (classical) Fabry disease (FD). No randomized clinical trials comparing effectiveness of different pain management strategies have been ...

Sangamo Therapeutics Inc (SGMO) reports significant milestones and strategic initiatives, despite financial challenges, in ...

We also support scientific advancement through targeted grant programmes, such as findforrare.com, to ensure ongoing research in Fabry disease, alpha‐mannosidosis, and nephropathic cystinosis.

Announced derisking milestones in pathway to anticipated biologics license application (BLA) submission for isaralgagene civaparvovec in Fabry disease, including all patients having passed one-year ...

Glycosyrin represents a novel type of "iminosugars," many of which are used as human drugs to treat metabolic disorders such as type-II diabetes and Fabry disease, because they are stable and ...

Our vision is to take this product from Argentina to the world.” Treatments for Fabry disease are considered high-cost, often posing a challenge to the sustainability of healthcare systems and ...

This particular candidate is being developed to treat patients with Fabry Disease. A good update was also provided here a few months ago, whereby the independent data monitoring committee (IDMC ...