As communicated by Roche, the first site to run the Rainbowfish clinical trial had been activated and is ready to accept presymptomatic babies diagnosed with spinal muscular atrophy.

In the Rainbowfish trial, doctors study the safety and efficacy of the oral drug risdiplam to treat newborns with SMA, in the hope that it will altogether prevent the appearance of symptoms.

The trial will enrol children younger than 6 weeks who have been diagnosed with spinal muscular atrophy but who do not yet display the symptoms of the disease. They will all receive risdiplam for 24 months, during which they will be monitored for improvements and any side effects. After successful completion of the 24-month study, all children will be invited to take part in open-label extension trial in which they will receive risdiplam for another 3 years.

The active trial site is in Gdansk, Poland. Other sites in Australia, Belgium, Brasil, Italy, Russia and USA are to be activated in the next few months.

This is the third clinical trial in which presymptomatic treatment of spinal muscular atrophy is investigated. In the ongoing Nurture trial, 25 SMA newborns have been presymptomatically treated with nusinersen (Spinraza) and now, three years later, show incredible results. In 2018, a new presymptomatic trial of AVXS-101 gene therapy opened in Liege, Belgium and more recently in London. Now, risdiplam join the bandwagon with another trial at new trial sites.

It is now crucial that all newborns nationwide are routinely screened for spinal muscular atrophy and offered treatment options. Unfortunately, the NHS recently concluded that newborn screening for SMA should not be commissioned in the UK. We are contesting this recommendation, because early treatment yields best results.

Parents who are expecting a baby with spinal muscular atrophy are invited to contact TreatSMA to discuss the options.