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

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) ...

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

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 ...

LEXINGTON, Mass. - uniQure N.V. (NASDAQ: NASDAQ:QURE), a gene therapy company with a market capitalization of $767 million, ...

PRINCETON, N.J., Feb. 03, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that two oral ...

Sangamo (SGMO) reported positive updated data from a Phase 1/2 study of its gene therapy candidate ST-920 in the treatment of Fabry disease. Read more here.

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 ...

The global enzyme replacement therapy market size was valued at USD 10.12 billion in 2024 and is projected to grow from USD 11.35 billion in 2025 to reach USD 21.29 billion by 2033, exhibiting a CAGR ...

Qure (QURE) announced the completion of enrollment in the first cohort of the Phase I/IIa trial of AMT-191, an investigational gene therapy ...