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.
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.
NICE has today (29 October 2020) announced that it has begun the process to review data collected as part of the Managed Access Agreement (MAA) for nusinersen.
Larotrectinib (also called Vitrakvi and made by Bayer), is a new treatment for a range of cancers, has been recommended for use in the Cancer Drugs Fund (CDF).
A paper published and co-authored by NICE staff looks at how histology-independent cancer drugs might be appraised.
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