CALD

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FDA restricts use of bluebird's Skysona gene therapy

The FDA has added a warning label to bluebird bio's Skysona, after launching a probe into cases of blood cancer linked to the gene therapy.

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).

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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

News

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.

News

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

FDA restricts use of bluebird's Skysona gene therapy

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

NHS staff 'feel unprepared for digital transformation'

How to meet your physicians where they are online

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