gene therapies

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Ultragenyx gets FDA nod for first Sanfilippo A gene therapy

Patients in the US with Sanfilippo syndrome type A have their first FDA-approved therapy, Ultragenyx Pharma's $3.9m one-shot gene therapy Fayuvi.

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Novartis' new SMA gene therapy cleared in EU

Novartis has secured EU approval for Itvisma, a new formulation of its spinal muscular atrophy gene therapy usable in a broader patient population.

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Vertex gets paediatric FDA okay for Casgevy gene therapy

For the first time, children in the US as young as two with blood disorders sickle cell disease and beta thalassaemia are eligible for gene therapy.

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uniQure sets course to US filing for Huntington gene therapy

Shares in uniQure have rocketed this morning, as it emerges that the FDA will not require it to run a new trial of Huntington's gene therapy AMT-130.

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Touchdown for Rocket as its first gene therapy gets US okay

Rocket Pharma has won FDA approval for the first gene therapy for LAD-I, a devastating genetic disorder causing severe vulnerability to infections.

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Boy in a wheelchair looking out to the forest

Regenxbio, Solid report data with Duchenne gene therapies

Regenxbio and Solid Bio say their gene therapies for Duchenne muscular dystrophy show no sign so far of the liver issues seen with Sarepta's Elevidys.

Ultragenyx gets FDA nod for first Sanfilippo A gene therapy

Novartis' new SMA gene therapy cleared in EU

Vertex gets paediatric FDA okay for Casgevy gene therapy

uniQure sets course to US filing for Huntington gene therapy

Touchdown for Rocket as its first gene therapy gets US okay

Regenxbio, Solid report data with Duchenne gene therapies

Lily gets FDA nod for SERD-CDK 4/6 combo in breast cancer

Discover a new era of healthcare panel engagement: Grounded ...

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