Mutations in this gene cause Gaucher disease. The drug restores the function of the defective GBA1 gene and alleviates the condition. Six patients have been dosed in the study and two of those ...

Patient advocacy groups warned that delays in implementing India's rare disease policy have left many, including children, in ...

Together the organisations are developing algorithms for identifying Fabry and Gaucher patients through ... with Microsoft and European rare disease patient alliance Eurordis on the Global ...

Gaucher is a genetic disease where glucocerebroside accumulates ... The European orphan drug patient group, Eurordis, has called for pharma to develop more rare disease drugs, but at a lower ...

The government may slash duties on imported rare disease and cancer drugs in the Union budget aiming to make lifesaving ...

Additional details on the meeting can be found at the WORLD Symposium website. About Spur Therapeutics Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing ...

While each genetic disease may occur in a small population of patients, collectively rare ... treat some inborn errors of metabolism, such as Gaucher disease). Now, gene therapy offers the ...

The list includes primary immunodeficiency disorders, Familial Duchenne Muscular Dystrophy (DMD), Familial Hypercholesterolemia, Spinal Muscular Atrophy (SMA), Gaucher’s Disease, Pompe Disease ...

The therapy is a critical treatment option for various life-threatening disease. Earlier, GST on gene therapy was set at 12 ...