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New drug for Spinal Muscular Atrophy patients

 Melvil Vedrenne-Cloquet
Melvil Vedrenne-Cloquet Image credit:

A new drug could change the lives of people living with a condition which causes weakness in the muscles and genetic death in young children.

It’s estimated there’s around 1,500 people living with Spinal Muscular Atrophy (SMA) who would benefit from a syrup known as Risdplam.

NHS England chief executive, Amanda Pritchard, said: “In the last three years the NHS has revolutionised care for people with SMA, by securing access to a trio of innovative treatments – Spinraza, Zolgensma and now risdiplam – where three years ago clinicians had no effective medicines at all.

“Spinal muscular atrophy is a cruel disease and the leading genetic cause of death among babies and young children, which is why NHS England has been determined to make these treatments available to people as soon as possible to help transform the lives of patients and their families.”

The drug will be available for only people with limited types of SMA including new born babies after the NHS managed to agree a discounted price for risdplam which usually costs £7,900 per 80ml, a price set by its manufactures Roche.

Dr Elizabeth Wraige, a consultant paediatric neurologist at Evelina London children’s hospital, part of Guy’s and St Thomas’ NHS foundation trust, welcomed the news.

She said: “This will be especially important for those with SMA who cannot receive either of the two existing treatments, Spinraza and Zolgensma. These are very exciting times and I am sure this news will be welcomed by those families and individuals affected by SMA as well as by their clinicians.”

The deal was made between NHS England, Roche and the National Institute for Health and Care Excellence (Nice).

Meindert Boysen, deputy chief executive of Nice, said: “This will not only be less burdensome, and therefore have a positive impact on the lives of both people with SMA and their caregivers, but it will also reduce the treatment administration requirements for the NHS.

“In practical terms, the availability of an oral drug should lead to greater adherence to treatment, along with giving access to a treatment to those who aren’t able to have other currently recommended options.”

One person who appreciates how beneficial risdplam can be is 9-year-old Melvil Vedrenne-Cloquet (pictured above) who started taking the drug when he was five.

His mum, Eve Chirdkiatisak, told The Guardian: “It’s fantastic for the other children and their families out there that it will now be available on the NHS, because when it comes to a diagnosis of SMA, time is of the essence. Children will never get what they have lost back, so the faster they can get the right medicines the better.”

For advice and support on Spinal Muscular Atrophy please visit the Spinal Muscular Atrophy UK website.