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

Allogeneic hematopoietic stem cell transplantation (HCT) has been shown to have disease-modifying effects in the demyelinating cerebral form of X-linked adrenoleukodystrophy (X-ALD), but finding a ...

The FDA last week announced that it is looking into the safety of bluebird bio ’s gene therapy Skysona (elivaldogene ...

While the use of elivaldogene autotemcel (eli-cel; Skysona) gene therapy has demonstrated long-term efficacy in patients with ...

The agency is also evaluating if there's a need for further regulatory action based on reports of hematologic malignancies from clinical trials of the therapy.

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 ...

The U.S. Food and Drug Administration is weighing the need for regulatory action on bluebird bio's gene therapy for a rare ...

Gene therapies have already been tested for other forms of ALD, which include cerebral adrenoleukodystrophy (CALD), a severe form of a rare inherited neurological disease, as well as adrenal ...

Data show SBT101 effectively delivered a functional copy of the ABCD1 gene to spinal cord tissues with resultant protein expression, addressing the underlying cause ...

Share on Facebook. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The FDA said on Wednesday that it is investigating reports ...

Thus, CD36 is uniquely positioned at the crossroads of VLCFA uptake and regulation of the inflammatory response and may impact ALD pathology through either of these mechanisms. More work is ...