Fabry's disease is an inherited condition that develops due to a mutation in the GLA gene. For most patients with this disease, the mutation causes the body to produce insufficient amounts of ...

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.

As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...

Sangamo Therapeutics Inc (SGMO) reports significant progress in its Fabry disease program and neurology therapies, while ...

Reports Q4 revenue $7.6M, consensus $11.7M. “I am pleased with Sangamo’s (SGMO) pipeline progress since the start of 2024. We advanced our two ...

Italian pharma Chiesi has begun a new rare diseases division as it builds towards a potential commercial launch for its Fabry Disease therapy. Chiesi, which is best known for its respiratory drugs ...

Amicus Therapeutics benefits from strong revenue and limited competition. Learn why FOLD stock is a BUY, with a favorable ...