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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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A giemsa stained blood smear from a person with beta thalassaemia

First CRISPR drug Casgevy gets swift second approval

The FDA has lost no time in approving Vertex Pharma and CRISPR Therapeutics pioneering gene-editing therapy Casgevy for its second use, approving the drug for transfusion-

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Vertex Pharmaceutical's Boston headquarters

FDA clears two sickle-cell gene therapies, including CRISPR ...

The US FDA has cleared two novel gene therapies for sickle cell disease: Vertex Pharmaceuticals' Casgevy and bluebird bio's Lyfgenia.

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FDA sets Dec deadline for Vertex, CRISPR sickle cell therapy

The FDA has started a priority review of Vertex Pharma and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel) in sickle cell disease (SCD), with a decision on approva

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ICER backs sickle cell gene therapies at one-off cost of $2m

The influential Institute for Clinical and Economic Review (ICER) in the US has issued draft guidance suggesting that new gene therapies for sickle cell disease (SCD) from

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bluebird unveils $2.8m price for gene therapy Zynteglo on FD...

bluebird bio's Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require re

New film aims to raise awareness of rare disease cALD

First CRISPR drug Casgevy gets swift second approval

FDA clears two sickle-cell gene therapies, including CRISPR ...

FDA sets Dec deadline for Vertex, CRISPR sickle cell therapy

ICER backs sickle cell gene therapies at one-off cost of $2m

bluebird unveils $2.8m price for gene therapy Zynteglo on FD...

Lexicon's type 1 diabetes drug voted down by FDA adcomm

Global reach, personal touch: Mastering customer engagement ...

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