Gaucher disease (GD) is an inherited disorder caused by recessive mutations in the GBA1 gene that encodes the lysosomal enzyme β-glucocerebrosidase (β-GC). β-GC hydrolyzes glucosylceramide (GluCer) ...

Importance: Gaucher disease (GD) is a rare lysosomal storage disorder with limited treatment options for pediatric patients. Oral substrate reduction therapy (SRT) with eliglustat offers a potential ...

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 ...

The data displayed for this chart goes back to 1994, however we hope to be able to offer deeper historic information at a future point ...

The Global Recombinant Chemicals Market is valued at approximately USD 2.9 billion in 2023 and is anticipated to grow with a healthy growth rate of more than 7.8% over the forecast period 2024-2032.

So those following the field know that there have been g cases developed peripherally for the treatment of Gaucher disease, but there’s also central involvement, not only in Gaucher disease ...

June 10, 2024 — Scientists have grown 'mini-guts' in the lab to help understand Crohn's disease, showing that 'switches' that modify DNA in gut cells play an ... Apr. 24, 2024 — Study ...

After hours: March 21 at 7:51:43 PM EDT Loading Chart for PLX ...

The data displayed for this chart goes back to 1997, however we hope to be able to offer deeper historic information at a future point ...

Alector at TD Cowen Conference: Advancing Neurodegenerative Therapies ...