NICE recommends first licenced treatment for AL amyloidosis
Having been available in Scotland and Northern Ireland since 2022, NICE’s positive opinion of the combination treatment means it is now accessible on the NHS.
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Having been available in Scotland and Northern Ireland since 2022, NICE’s positive opinion of the combination treatment means it is now accessible on the NHS.
In this exclusive article, Biogen’s Vice President and Head of the Neuromuscular Development Unit, Dr Toby Ferguson, discusses the current rare disease landscape and recent developments in treating Friedreich’s ataxia (FA).
Advanced therapy medicinal products (ATMPs) will be among the first to undergo joint clinical assessments at EU level in 2025 and these will form the basis for national value assessments and pricing negotiations. Here, Paolo Morgese from the Alliance for Regenerative Medicine discusses how methodologies for joint clinical assessments (JCAs)…
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