“Diagnosing Gaucher disease typically involves a combination ... thereby preventing its accumulation in cells. It is typically used for patients who cannot undergo ERT or for those with milder ...

Evidence suggests that in Parkinson’s disease, a breakdown in lysosomal function allows lipids to build up within cells. Variants of the GBA1 gene, the strongest genetic risk factor for sporadic PD, ...

Gaucher disease requires lifelong treatment ... Waiting queue growing longer According to PGIMER’s Poor Patient Cell, 193 patients are waiting for the grant to start their treatment.

In this webinar – ‘Enabling Digitally Enhanced Care for Rare Diseases in Europe: Integrating AI and Connected Care ...

Gaucher's disease is caused by a deficiency ... Both drugs are formulations of α-galactosidase, but are produced in different cell lines. In the United States, Genzyme beat TKT and garnered ...

The industry is looking, with renewed hope, to the “promise” of messenger RNA (mRNA) therapeutics for a wide range of diseases beyond COVID-19, and not only in vaccine form but also for gene and cell ...

12 lac patients affected Diseases such as haemophilia, thalassemia, Pompe disease, Gaucher disease, and sickle cell anaemia are categorized as rare diseases, most of which are genetic. While the exact ...