Exagamglogene autotemcel for the treatment of patients with sickle cell disease

14 March 2024 - The Department of Health and Social Care has asked the NICE to produce guidance on the ...

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Etranacogene dezaparvovec for the treatment of patients with moderately severe or severe haemophilia B

2 August 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...

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Onasemnogene abeparvovec for the treatment of patients with presymptomatic spinal muscular atrophy

19 April 2023 - NICE has published Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for the treatment of babies aged 12 months ...

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Eladocagene exuparvovec for the treatment of patients with aromatic L-amino acid decarboxylase deficiency

19 April 2023 - NICE has published evidence based recommendations on eladocagene exuparvovec (Upstaza) for the treatment of patients 18 ...

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NICE recommends life-changing gene therapy for children with ultra rare genetic disorder in final draft guidance

23 March 2023 - The first and currently only gene therapy for children with an ultra-rare genetic disorder has been recommended ...

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UK’s most expensive drug Libmeldy saved Teddi Shaw, but is too late for her sister

16 February 2023 - A toddler with a rare inherited condition has become the first child to be treated by ...

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Vutrisiran for the treatment of patients with hereditary transthyretin-related amyloidosis

15 February 2023 - NICE has published final draft guidance on the use of vutrisiran for the treatment of adults ...

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NICE backs Alnylam’s Amvuttra for rare form of amyloidosis

19 January 2023 - Alnylam’s gene-silencing drug Amvuttra has been recommended as a treatment for hereditary transthyretin-related amyloidosis by NICE, ...

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Atidarsagene autotemcel for treating metachromatic leukodystrophy

28 March 2022 - NICE has published evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children. ...

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First gene therapy for rare genetic neurodegenerative disorder in children, recommended in NICE draft guidance

4 February 2022 - Children with a rare, fatal, genetic disorder will be able to benefit from a new one-off treatment ...

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Libmedly: world's 'most expensive' drug recommended for NHS use

4 February 2022 - The NHS has struck a confidential deal for what's thought to be the most expensive drug ...

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NICE recommends gene silencing therapy for porphyria patients on NHS

21 October 2021 - NICE have recommended the use of givosiran, a gene silencing therapy, on the NHS in England, as ...

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Implementing outcomes based managed entry agreements for rare disease treatments: nusinersen and tisagenlecleucel

10 August 2021 - Enthusiasm for the use of outcomes based managed entry agreements to manage uncertainties apparent at the time ...

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Colchester baby's parents feel 'abandoned' over £1.7 million drug

25 May 2021 - A baby with a fatal genetic condition has been "abandoned" in a "race against time" for ...

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NICE announces more people eligible for nusinersen sodium following review of Managed Access Agreement

4 May 2021 - NICE has today announced that more people with the rare genetic disorder spinal muscular atrophy are to ...

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