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 ...

The company said that recently announced partnerships with Roche's Genentech unit and Astellas, and advanced business development discussions for its Fabry gene therapy programme (isaralgagene ...

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 ...

Qure's long-term success hinges on overcoming regulatory and logistical challenges. See why I rate QURE stock a hold.

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 ...

And our Fabry gene therapy study continues to generate best-in-class data, with a pivotal data readout expected in mid-2025.

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 ...

Figure 1: Enhancement of α–Gal A in lymphoblasts from patients with Fabry disease. Figure 2: Effect of DGJ on expression of α–Gal A in normal and mutant cells. To evaluate intracellular ...

In plants, the space between cells is a key battleground during infection. To avoid recognition in this space, a strain of ...

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