X-linked adrenoleukodystrophy (X-ALD) is a debilitating disease caused by mutations in the ABCD1 gene. Approximately 30% of affected boys develop the progressive ...

The work of Professor Fanny Mochel (AP-HP, Sorbonne University) at Paris Brain Institute, in collaboration with clinical research teams in eight countries and Spanish biotech Minoryx Therapeutics, has ...

KUTV — The Utah Department of Health’s Newborn Screening (NBS) program has added a test for all babies born in the state that detects a debilitating genetic disorder. On Sept. 20, 2020, the NBS ...

Please provide your email address to receive an email when new articles are posted on . Most patients who received Skysona for cerebral adrenoleukodystrophy did not have major functional disabilities ...

Please provide your email address to receive an email when new articles are posted on . Biotechnology company bluebird bio announced that an FDA advisory committee has unanimously endorsed its gene ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--NeuroVia, Inc., a biopharmaceutical company focused on developing innovative therapies for rare genetic neurological diseases, announced the closing of a Series A ...

Cerebral adrenoleukodystrophy (CALD) is a rare progressive, genetic brain disease that primarily presents in young boys, causing loss of neurological function and ultimately leading to early death.

VANCOUVER — Autologous hematopoietic stem cell (HSC) gene therapy may offer a safe and effective alternative to allogeneic bone marrow transplant for patients with cerebral adrenoleukodystrophy, ...