AceLink Therapeutics, Inc., a clinical-stage biotech company developing next-generation oral substrate reduction therapies (SRTs), presented interim d ...

RICHMOND, Calif. - Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company with a market capitalization of $263 million, has announced promising updated results from its Phase 1/2 STAAR ...

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 ...

Methods: Data from medical records of the 44 heterozygous females followed at Cedars-Sinai Medical Center were compiled and analyzed for symptoms of Fabry disease. Quality of life data were also ...

Sangamo (SGMO) announced updated data from the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene ...

This clinic started due to a specific Rare Genetic Kidney disease called Fabry Disease. Fabry Disease affects 1:40,000 individuals. This clinic is one of the country’s largest Fabry Disease clinics ...

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.

Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions ...

PLX READ THE FULL PLX RESEARCH REPORT Reasons To Own / A Compelling Idea for 2025 Protalix Biotherapeutics, Inc. (NYSE:PLX) ...

The firm will begin testing a second dose cohort with AMT-191 after receiving a positive recommendation from a data monitoring committee.

PRINCETON, N.J., Feb. 03, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that two oral presentations and 20 posters across its development programs will be included at ...