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 ...

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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 ...

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

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 ...

driven by new oral drugs such as Galafold as well as gene therapies that are still in clinical development. It says ERTs will remain a key part of Fabry treatment, although the first generation ...

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.