Of the 506 patients listed on the national crowdfunding portal, 242 suffer from Gaucher. Of these, 68 patients are currently receiving treatment while 128 are on the waitlist. Additionally ...

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 ...

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

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

Gaucher disease (GD; MIM230800 ... Accumulation also occurs in the central nervous system (CNS) in some patients causing progressive neurological manifestations, resulting in neuronopathic GD (nGD).1 ...

Background: Gaucher disease is classified into neuronopathic and non-neuronopathic forms with wide phenotypic variation among patients sharing the same genotype. While homozygosity for the common ...

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 ...