has reached a significant milestone in the development of its gene therapy product candidate for Fabry disease, isaralgagene civaparvovec, also known as ST-920. According to InvestingPro data ...

Sangamo Therapeutics, Inc. , a genomic medicine company, today announced important derisking events in the pathway to a planned BLA submission for isaralgagene civaparvovec, or ST-920, its wholly ...

Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is ...

Qure to host inaugural earnings call on Friday, May 9, 2025 at 8:30 a.m. ET ~LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 05, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading ...

Developments in genomics are enabling the rise of personalised medicine, with therapies tailored specifically for individuals ...

Galafold (migalastat) is an oral precision medicine for adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant, based on in vitro assay data.

Qure to host inaugural earnings call on Friday, May 9, 2025 at 8:30 a.m. ET ~LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 05, 2025N.V. (NASDAQ: QURE), a leading gene therapy company advancing ...

A "productive" meeting with the FDA has given the firm confidence in its regulatory filing and commercialization plans for ST-920.

which is the development of gene therapy AMT-191. This particular candidate is being developed to treat patients with Fabry Disease. A good update was also provided here a few months ago ...

MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD ...