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

Spur Therapeutics has announced that its gene therapy candidate for Gaucher disease will soon be moving to a Phase III trial after positive early data from a Phase I/II trial. Spur, which is a ...

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 ...

Sanofi is still running studies of the glycosphingolipid (GSL) inhibitor in rare lysosomal storage diseases like Gaucher disease type 3, Fabry disease and GM2 gangliosidosis (also known as Tay ...

After completing medical school at Tulane University and a pediatric residency at Northwestern University, Sidransky in 1988 undertook a medical genetics fellowship at the NIH and started studying ...

Later, in 1951, while working with Dr. Sidney Farber, professor of Pathology at Harvard, Dr. Uzman isolated and described the lipoprotein in Gaucher's disease, an anemia of the spleen that runs 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 ...

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 ...

About 3,000 people in Singapore have been diagnosed with rare diseases such as bile acid synthesis disorder, Gaucher disease and Pompe disease, and around 700 of them are children. Most cases are ...