Rare diseases

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AstraZeneca buys rare disease firm Amolyt for $1.05bn

AstraZeneca has added a late-stage rare disease therapy to its pipeline, buying France’s Amolyt Pharma and its hypoparathyroidism drug eneboparatide for $800 million upfro

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NICE no follows Scottish aye for rare disease drug Xenpozyme

NICE has turned down Sanofi’s Xenpozyme as a treatment for the rare disease acid sphingomyelinase deficiency (ASMD), creating a situation where access to the drug in the U

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RNA therapy firm Avidity files $400m placement

Avidity Biosciences has filed a private placement worth around $400 million, seeking a cash infusion to help advance its pipeline of clinical-stage candidates that combine

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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

In U-turn, NICE backs first drug for AL amyloidosis

Almost four years after it was approved in Europe to treat the incurable rare disease systemic amyloid light-chain (AL) amyloidosis, Johnson & Johnson’s Darzalex has b

News

Clinigen educates on early access to rare disease therapies

On Rare Disease Day, Clinigen has launched a campaign to try to encourage doctors and patients to explore how they can get early access to investigational therapies.

AstraZeneca buys rare disease firm Amolyt for $1.05bn

NICE no follows Scottish aye for rare disease drug Xenpozyme

RNA therapy firm Avidity files $400m placement

New film aims to raise awareness of rare disease cALD

In U-turn, NICE backs first drug for AL amyloidosis

Clinigen educates on early access to rare disease therapies

Heart disease risk ‘calculator’ could save lives

Fixing clinical trial data management

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