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 ...

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 ...

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.

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

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 ...

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.

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 ...

For informational purposes only. Consult a medical professional for advice. Source: Focus Medica. A genetic disorder characterized by damage to nerve tissue, and adrenal gland dysfunction.

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