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Showing 1 to 6 of 6 results for exagamglogene autotemcel
Evidence-based recommendations on exagamglogene autotemcel (Casgevy) for transfusion-dependent beta-thalassaemia in people 12 years and over.
Evidence-based recommendations on exagamglogene autotemcel (Casgevy) for treating severe sickle cell disease in people 12 years and over.
Managed access allows people to access promising new treatments that would otherwise not be recommended because it is too uncertain whether the treatment is cost-effective.
This document describes a real-world evidence framework that aims to improve the quality of real-world evidence informing our guidance. The framework does not set minimum standards for the acceptability of evidence. The framework is mainly targeted at those developing evidence to inform NICE guidance. It is also relevant to patients, those collecting data, and reviewers of evidence
NICE approves groundbreaking one-off gene therapy for severe sickle cell disease
People in England with severe sickle cell disease will be among the first to receive treatment using revolutionary CRISPR gene editing technology, following publication of our final draft guidance today.
Patients in England with severe beta-thalassaemia will be amongst the first in Europe to benefit from one-time gene therapy exagamglogene autotemcel.