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 ...
The following is a summary of “Cutaneous manifestations of Fabry disease: A systematic review,” published in the March 2025 ...
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.
Sangamo Therapeutics Inc (SGMO) reports significant progress in its Fabry disease program and neurology therapies, while ...