Sangamo Therapeutics Announces Important Derisking Milestones in Pathway to Anticipated BLA Submission for ST-920 in Fabry Disease ...

4D Molecular Therapeutics (4DMT) has said a trial of its gene therapy for Fabry disease has been placed on a clinical hold by the FDA, a few weeks after it reported three cases of atypical ...

This would be in terms of the development of its Fabry Disease program, which uses gene therapy isaralgagene civaparvovec to treat these patients. The reason why investors should care about this ...

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.

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 ...

is leading the gene therapy study in Calgary.Dr. Jeffrey Medin, Affiliate Scientist at UHN, and MACC Fund Professor at the Medical College of Wisconsin, pioneered the Fabry gene therapy treatment ...

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 ...