Gaucher disease

May 21, 2025

Lysosomal Storage Disorders, or LSDs, are rare genetic conditions that may not make headlines, but they affect thousands worldwide, often with life-changing consequences. LSDs happen when the body’s lysosomes, tiny cellular recycling centers, fail to break down waste materials due to missing or faulty enzymes. This...

May 07, 2020

Avrobio taps ADC of Magenta for enhancing gene therapy conditioning Avrobio is working to make conditioning safer. The company is collaborating with Magenta Therapeutics to check antibody-drug conjugate. Under the agreement, the partners will evaluate Magenta’s lead conditioning program, MGTA-117, with at lea...

Dec 11, 2019

Gaucher Disease Market is anticipated to enhance in the forecast period owing to a strong Gaucher disease pipeline. According to the National Organization of Rare Diseases, Gaucher disease is a rare, inherited, metabolic disorder, which is characterized by an accumulation of harmful fats, chiefly the glycolipid ...

Dec 11, 2017

Gaucher Disease is a rare autosomal recessive inherited multisystem disease with phenotypic variation from mild to very severe. It is the most common lysosomal storage disorder which occurs due to the deficiency of an enzyme β-glucocerebrosidase that helps in degrading glucocerebroside into glucose and ceramide. Lac...

Oct 13, 2015

Introduction: Any disease that affects less than 200,000 patients or 1 in 1500 is termed as a rare disease. There are around 5000 to 8000 rare disease reported, out which only 10% have cure. According to the European Organization for rare disease, 80% of rare diseases are genetic in nature. Some of the rare disease...