In college, I did not feel good at all in my body. I knew something was wrong, and I wanted to get to the bottom of it. I would take a quick trip to the grocery store, leave exhausted, and come home ...

Gregory and Deborah Macres from California lost their son, Gregory, in 1997, when he was just 4 years old. Little Gregory had a severe form of a rare genetic disorder called Gaucher disease and died ...

People with the rare inherited metabolic disorder Gaucher disease have a deficiency in the lipid-digesting glucocerebrosidase enzyme, which causes the accumulation of harmful levels of glucolipids in ...

Single dose of avigbagene parvec (FLT201) demonstrates sustained clinical benefit and favorable safety up to two years after withdrawal of prior ...

LONDON, May 27, 2025 (GLOBE NEWSWIRE) -- Spur Therapeutics today announced the publication of preclinical proof-of-concept data for FLT201, its adeno-associated virus (AAV) gene therapy candidate for ...

Lysosomal homeostasis is crucial to the metabolism of certain proteins and lipids that would otherwise accumulate, thus leading to cellular stress and pathology. This is common in diseases such as ...

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 ...