SynaptixBio announces milestone with selection of candidate drug to treat rare disease H-ABC: Our Bureau, Bengaluru Friday, June 20, 2025, 12:30 Hrs [IST] Oxford-based SynaptixBio ...

Leukodystrophy isn’t just one disease; it’s actually a group of diseases that affect the central nervous system. Doctors are discovering new forms of leukodystrophy all the time, but experts ...

SynaptixBio announces a major milestone with SB H-19642 in the upcoming clinical trial for treating H-ABC disease.

Also known as globoid cell leukodystrophy, Krabbe disease is classified ... In Krabbe disease, the lack of an enzyme in the lysosome causes a toxic buildup that leads to the destruction of myelin.

Myelin is the protective covering of the nerve that helps signals travel through the nerve. There are over 40 known types of leukodystrophy, each affecting different parts of the myelin, causing a ...

Metachromatic leukodystrophy, MLD for short, is a rare but highly debilitating neurodegenerative disease. It is noteworthy for its difficulties and the potential promise the latest research offers ...

When that happens, proteins and other substances build up in cells and become toxic. They can damage cells and organs in the body. This article covers some of the most common lysosomal storage ...

Please provide your email address to receive an email when new articles are posted on . Biotech firm SynaptixBio has entered into a licensing agreement with the Children’s Hospital of ...

The Food and Drug Administration on Monday approved a gene therapy for metachromatic leukodystrophy, a devastating genetic disorder that eats away at affected children’s neurons. The medicine ...

Metachromatic leukodystrophy is an incurable rare genetic disease. At 5 years of age, 71% of children given Lenmeldy were able to walk without assistance, and 85% had normal language scores.