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Showing 1 to 9 of 9 results for onasemnogene
Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy (HST24)
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating presymptomatic spinal muscular atrophy in babies aged 12 months and under.
Onasemnogene abeparvovec for treating spinal muscular atrophy (HST15)
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies.
In development Reference number: GID-TA11733 Expected publication date: 26 August 2026
Nusinersen and risdiplam for treating spinal muscular atrophy (review of TA588 and TA755) [ID6195]
In development Reference number: GID-TA11386 Expected publication date: TBC
Evidence-based recommendations on risdiplam (Evrysdi) for 5q spinal muscular atrophy (SMA) in people of all ages.
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency (HST26)
Evidence-based recommendations on eladocagene exuparvovec (Upstaza) for treating aromatic L-amino acid decarboxylase deficiency in people 18 months and over.
Our forward view highlights the topics we will prioritise in the coming year.
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 life-changing gene therapy for treating spinal muscular atrophy
A new one-off gene therapy for babies with the rare genetic disorder spinal muscular atrophy (SMA) is set to become the most expensive treatment ever approved by NICE.