NICE recommends Sprinraza for 5q spinal muscular atrophy

The recommendation is for infants, children and adults with 5q spinal muscular atrophy and will be available through a scheme known as a Managed Access Agreement (MAA), meaning that patients will be able to get Spinraza while more long-term data on its efficacy is gathered.

The evaluation based on the largest body of clinical evidence currently available across all types of SMA, with six years of data including more than 300 patients treated with pre- symptomatic, infantile and later-onset, or types I and II SMA.

Muscular Dystrophy UK and Spinal Muscular Atrophy UK are now calling on NICE to formally recommend the treatment and for Biogen and NHS England to urgently implement the MAA so patients can access the drug quickly, and for Wales and Northern Ireland, which generally follows NICE’s guidance, to follow suit.

The announcement is "fantastic news that gives families home", said Catherine Woodhead, chief executive of Muscular Dystrophy UK.

She continued, "Children already receiving treatment are reaching milestones never thought possible and living longer, and now hundreds of others will be given that same chance.

"This would not have been possible without the support and hard work of clinicians, SMA charities, MPs and, above all, families and people with the condition. Thanks to all of us working together and our tireless campaigning, patients should face a brighter future."

Currently, patients in 24 European countries - and many more around the world - have access to the treatment via regular reimbursement, with most countries making it available in order to treat a broad range of patients.

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