BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)--CANbridge Pharmaceuticals Inc. (HKEX:1228), a China and US-based global biopharmaceutical company committed to the research, development and ...
New compelling clinical data from first-ever Gaucher disease type 3 (GD3) patient and four Gaucher disease type 1 (GD1) patients dosed with investigational AVR-RD-02 Data from first pediatric GD3 ...
As gene therapies ramp up in spinal muscular atrophy and hemophilia, it may soon be the turn of Gaucher disease patients to realize the latent potential of this type of treatment. That’s according to ...
A rare genetic disorder that disproportionately affects Ashkenazi Jews is also a safeguard against tuberculosis, a new study shows, and that discovery could explain a longstanding scientific mystery.
A new treatment may be on the horizon for a rare genetic disease that disproportionately affects Ashkenazi Jews. For years, the most common treatment for Type 1 was enzyme replacement therapy, but ...
A study by researchers at Baylor College of Medicine, the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital and collaborating institutions is the first to associate ...
Family members who care for patients with Gaucher disease (GD) reported lower health-related quality of life (HRQOL) scores than the general population in every area of an online survey, according to ...
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