Tay-Sachs disease is a rare hereditary disorder that occurs when the body lacks an enzyme that can break down fatty ...

In one particular variant of this disease known as mucopolysaccharidosis I (MPS I), a deficiency of the enzyme alpha-L-iduronidase causes a build up of GAGs in tissues and organs, which in turn ...

(RNS) — Some 25 years ago, a friend of mine and his wife whose little boy had been diagnosed with Tay-Sachs disease visited our home. A relatively rare genetic disorder that is less so among ...

Tay-Sachs disease results in the deterioration of the nervous system, usually resulting in death before the patient reaches age 5. It is caused by the absence of an enzyme which breaks down lipids ...

The heroes are voluntary organizations like the Wellcome Trust or the Committee for the Prevention of Jewish Genetic Disease, which has done much to eliminate Tay-Sachs disease and cystic fibrosis ...

Fabry disease and GM2 gangliosidosis (also known as Tay-Sachs disease), but the company's earlier assessment of venglustat as a "pipeline in a pill" is starting to look shaky. The phase 2/3 STAGED ...