Gaucher disease is a genetic disorder when the body is unable to break down certain fats, leading to fat accumulation in the ...

Gaucher disease is a rare inherited condition caused by the body’s inability to break down a fatty substance called glucocerebroside. This happens due to a lack of an important enzyme known ...

Bengaluru: The medical education department is reaching out to corporate entities for support in treating children affected ...

National Policy for Rare Diseases 2021 addresses diagnosis and treatment, categorizing diseases and advocating for affordable ...

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 ...

Kolkata: A three-year-old girl waiting for treatment for a rare disease died without getting any medical assistance under the ...

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 ...

that modify disease (for example, enzyme replacement therapy to treat some inborn errors of metabolism, such as Gaucher disease). Now, gene therapy offers the potential to address the underlying ...

Patients suffering from rare diseases are facing severe difficulties due to delays in receiving timely financial aid and ...

Infants with Type 2 Gaucher disease may experience symptoms such as poor muscle tone, seizures, and developmental delays, often leading to death at a young age. “Diagnosing Gaucher disease ...