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

These slow, agonizing deaths can be prevented—but our new science-averse health czar apparently doesn’t see that as a priority in his Make America Healthy Again crusade. Along with the 10,000 or so ...

The prevalence of ALD is estimated as between one in 20,000 and one in 50,000 individuals worldwide, and AMN accounts for around 25% to 46% of all ALD cases. Gene therapies have already been ...

The patients received the one-time autologous hematopoietic stem cell-based gene therapy as a treatment for early cerebral adrenoleukodystrophy (CALD) – a rare and fatal neurodegenerative disease.

US biotech bluebird bio has had a challenging time in the last few months, so a recommendation for EU approval of its gene therapy for adrenoleukodystrophy (ALD) will give it a lift. The EMA's ...