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 ...

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 ...

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 ...

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 ...

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 ...

The US Food and Drug Administration (FDA) has awarded breakthrough therapy designation to uniQure's AMT-130 for treating ...