Metachromatic leukodystrophy is a rare genetic disorder that mainly affects young children and results in severe neurological symptoms accompanied by a loss of motor and intellectual capacities ...

Neurology > General Neurology Pediatric Neuron Disease Gets Its First Gene Therapy — FDA approves arsa-cel for metachromatic leukodystrophy by Judy George, Deputy Managing Editor, MedPage Today ...

Each type of Leukodystrophy affects a different part of the myelin sheath, leading to a range of neurological problems. This includes issues with movement, vision, hearing, balance, the ability to ...

TUBB4a leukodystrophy is an "incurable genetic condition" that affects one in 1,663 births, according to the Cleveland Clinic. First identified in 2014, it makes up a small percentage of the 50 ...

DUBLIN--(BUSINESS WIRE)--Research and Markets has announced the addition of the "Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline Review, H2 2016" report to their offering. Globoid Cell ...

Release Therapeutics SA has announced that it has secured CHF3.3 million (US$3.87 million) in seed funding. The proceeds will be used to finance primate studies of the company’s cell ...

BEDFORD, Mass., Aug. 11, 2022 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the details of HMI-204, its optimized, in vivo, one-time ...