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

1. In this prospective cohort study, in boys with diagnosed cerebral adrenoleukodystrophy, it was found that lentiviral gene therapy containing ABCD1 complementary ...

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

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.

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

The FDA may have safety concerns abut bluebird bio's gene therapy for rare, fatal disease cerebral adrenoleukodystrophy (CALD), but its advisors believe its benefits far outweigh the risks ...

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 ...

Skysona is indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy.