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First UK clinical trial site for new combined SMA treatment

4 November 2022

Leeds Children’s Hospital will host the first UK clinical trial for a new treatment for children and young people living with spinal muscular atrophy (SMA).

The Phase 3 SAPPHIRE study aims to assess the safety and effectiveness of apitegromab used in combination with Spinraza (nusinersen) or Evrysdi (risdiplam) to treat children and young people aged 2-21 with type 2 or type 3 SMA who are unable to walk independently. 

Apitegromab selectively blocks the activation of a protein that restricts muscle growth called myostatin, which is found in skeletal muscles (that help us move and walk). Blocking the activation of myostatin may improve muscle and motor function in people living with SMA. 

“It’s encouraging to see this phase III clinical trial study that is combining the treatments Spinraza or Evrysdi, both of which are already available for people with SMA in the UK. Improving the muscle function in addition to restoring SMN protein could prove to be beneficial for people living with this condition.” Dr Kate Adcock, Director of Research, Muscular Dystrophy UK 

In September this year, Scholar Rock announced the opening of its first European sites for the study in Spain. They have now announced Leeds Children’s Hospital as the first trial site in the UK. 

Full inclusion and exclusion criteria are available at Please note that although the overall trial is recruiting for ages 2-21, the Leeds site will only be able to recruit children and young people who are aged 2-17 at date of screening.  

To express an interest in joining the trial, either for yourself or on behalf of your child, please contact the Leeds paediatric research team at 

Before registering for any clinical trials, always consult with your clinical team. Learn more about clinical trials.

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