News

The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. Risdiplam continues to show improvement or maintenance of motor function in people with Type 2 or Type 3 SMA

    Risdiplam continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with Type 2 or Type 3 SMAAs per our request to receive regular updates about the risdiplam clinical development programme, Roche has shared with us new 2-year longer-term data from part 2 of the SUNFISH clinical trial. These data will be presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference taking place from 15th to 18th March.

    SUNFISH part 2 is an ongoing, global study evaluating the efficacy and safety of risdiplam in people aged 2-25 years with Type 2, or non-ambulant Type 3 SMA. The study is placebo-controlled which means that patients were treated with risdiplam (n=120) or with placebo and risdiplam (n=60; patients in the placebo arm received placebo for 12 months followed by risdiplam treatment for 12 months).

    These new results of nearly a year of additional follow-up, show that people treated with risdiplam continue to demonstrate sustained benefit from the treatment. The natural history of the disease in people with Types 2 and 3 SMA who remain untreated decline in motor function over time. In more detail, gains in motor function seen after 12 months of treatment with risdiplam either continued to improve or were maintained at month 24 across a number of primary and secondary endpoint measures. In addition, the number of serious adverse events, high-grade adverse events and treatment-related adverse events observed decreased in the second year versus the first year, further demonstrating the favourable safety profile of risdiplam.

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