rare disease - Ataxia UK

rare disease

Ataxia UK and CureDRPLA provided feedback regarding the scope of a framework under development for individualised treatments in the UK

NHS England opened a consultation to seek feedback on an operational framework they are developing to support the future use of individualised genetic therapies in the NHS in England. They […]

Ataxia UK and CureDRPLA provided feedback regarding the scope of a framework under development for individualised treatments in the UK Read More »

MHRA publishes plans to reform medicines regulatory systems for rare diseases

Existing medicines regulatory processes do not consider the challenges that accompany treatment development for rare diseases. The regulatory body for medicines in the UK, the MHRA, has recognised this and is planning a major change by proposing a new framework for rare diseases. We welcome their report,

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