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

The hematology specialist officially diagnosed me with Gaucher disease. The bone marrow biopsy confirmed my diagnosis of ...

Find effective medications for 'Gaucher’s Disease treatment right here!' This page is a trusted source for both professionals and patients, detailing brand-name and generic options designed to ...

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

It acquired rights to taligurase alfa, a form of the enzyme glucocerebrosidase in development for the treatment of Gaucher's disease, from Protalix Biotherapeutics in Carmiel, Israel. Protalix has ...

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

England’s NHS will routinely fund Sanofi/Genzyme’s Cerdelga (eliglustat) for the rare condition Gaucher disease after a recommendation from NICE. The cost effectiveness body published final ...

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 ...

Genzyme anticipates supply constraints of Cerezyme (imiglucerase), a treatment for Gaucher disease, and Fabrazyme (agalsidase beta), used to treat Fabry disease, while the facility shuts down for ...

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, ...