Accumulation of fat molecules is detrimental to the cell. Researchers from the Yong Loo Lin School of Medicine, National ...

FDA aligned on single-arm Phase 3 study to support potential accelerated and full approvalOn track to dose first patient in Phase 3 trial in ...

New research has made a breakthrough in understanding how our cells manage to stay healthy by recycling important fat ...

Patients showed reductions in lyso-Gb1 levels, a biomarker of clinical response in Gaucher disease, ranging from 33% to 96%. Furthermore, the study observed maintenance of normal haemoglobin ...

Families of rare disease patients appeal to PM Modi and Health Minister for increased treatment funding support.

Results from GALILEO-1, a first in human clinical trial of FLT201 AAV-gene therapy in adult patients with Gaucher disease type 1 (Poster #318) Session: Poster Session III Date & Time: Thursday, ...

AceLink Therapeutics, Inc., a clinical-stage biotech company developing next-generation oral substrate reduction therapies (SRTs), presented interim d ...

Lyso-Gb1 is one of the best predictors of clinical response in Gaucher disease, and FLT201 showed rapid and sustained reductions in lyso-Gb1 in its Phase 1/2 GALILEO-1 trial. Full approval would ...

The FDA has offered positive feedback on possible paths forward for accelerated or traditional approval of FLT201 using data from a single-arm study.