CALD

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New film aims to raise awareness of rare disease cALD

Rare Disease Day saw the debut of a film that aims to raise awareness of cerebral adrenoleukodystrophy (cALD), a devastating and fatal condition with no pharmacological tr

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FDA panel backs bluebird's CALD gene therapy, despite safety...

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.

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Bluebird claims EU nod for rare disease therapy Skysona

Bluebird bio has its first approval for Skysona (formerly Lenti-D), its gene therapy for children with the rare disease cerebral adrenoleukodystrophy (CALD), after getting the nod from the

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How-cell-and-gene-therapies-are-transforming-the-pace-of-deal-making

CHMP backs bluebird bio's gene therapy for rare disease ALD

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.

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bluebird bio announces long-term data from CALD gene therapy

bluebird bio has announced new clinical trial data from its gene therapy for patients with cerebral adrenoleukodystrophy (CALD), including long-term follow-up data.

New film aims to raise awareness of rare disease cALD

FDA panel backs bluebird's CALD gene therapy, despite safety...

Bluebird claims EU nod for rare disease therapy Skysona

CHMP backs bluebird bio's gene therapy for rare disease ALD

bluebird bio announces long-term data from CALD gene therapy

Gene therapy firm Kate Tx snapped up by Novartis for $1.1bn

Coming soon: The Life Sciences Industry Report 2025 – insigh...

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