CALD

News

Refiling for cALD therapy on the cards after new data

Readout from NEXUS study sets up mid-2025 refiling for Minoryx and Neuraxpharm's leriglitazone for rare disease cerebral adrenoleukodystrophy (cALD).

News

FDA probing cancer cases with Bluebird's CALD gene therapy

The FDA is scrutinising reports of haematological cancers in patients treated with bluebird bio's Skysona gene therapy for neurological disease 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

News

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.

Refiling for cALD therapy on the cards after new data

FDA probing cancer cases with Bluebird's CALD gene therapy

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

World Without Disease initiative: 2024 update

The AI effect in pharma commercial strategy: From data to re...

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