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.

GD3 is a chronic neuropathic form of Gaucher disease characterized by progressive encephalopathy. The following article features coverage from the 17th Annual WORLDSymposium meeting. Click here to ...

This article was originally on a blog post platform and may be missing photos, graphics or links. See About archive blog posts. People who carry the gene for a rare genetic problem known as Gaucher ...

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 ...

ST. LOUIS--(BUSINESS WIRE)--M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage ...

LONDON, Oct. 01, 2025 (GLOBE NEWSWIRE) -- Spur Therapeutics today announced it will present new clinical data for avigbagene parvec (FLT201), its Phase 3-ready gene therapy candidate for Gaucher ...

Just five months old, Prajwal's life was turned upside down when his parents realised that his health was deteriorating. He had repeated high fevers, loose stools, and a swollen stomach, forcing his ...

Data highlight potential of FLT201 to set new standard of care for Gaucher disease Dramatic and durable reductions observed in lyso-Gb1, an established biomarker of clinical response, in patients with ...

LONDON, Oct. 07, 2025 (GLOBE NEWSWIRE) -- Spur Therapeutics today announced updated clinical data for avigbagene parvec (FLT201), its Gaucher disease gene therapy candidate, demonstrating clinical ...