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Page last updated: 2nd April 2024

The latest risdiplam (Evrysdi) data updates were presented at the 2023 MDA Clinical and Scientific Conference in March 2023 and at the SMA Europe Congress in February 2024.

March 2023:

Outcomes for those with SMA Type 2 or 3, aged 2 – 25 years

Patients enrolled via Part 1 of the trial

Patients involved in Part 1 continued to receive treatment at the dose selected from the 12-week dose-finding study.

  • Risdiplam significantly improved motor function after 48 months of risdiplam treatment:
    • MFM-32 (Motor function measure which assesses 32 items) total change from baseline was greater in patients receiving risdiplam compared with natural history data.

Patients enrolled via Part 2 of the trial

  • Risdiplam significantly improved motor function after 36 months of treatment:
    • The proportion of patients demonstrating a marked improvement (change ≥3 points) or stabilisation (change ≥0 points) in MFM-32 score were more likely in people who were on risdiplam than those in the untreated group.

February 2024:

Independent living and measuring what matters

Roche has developed the SMA Independence Scale-Upper Limb Module (SMAIS-ULM). This has been done in close partnership with CureSMA, SMAEurope, and the SMA Foundation. The aim is to measure the level of support needed to perform activities of daily living (ADL) and to better capture the patient and caregiver experience.

At the February 2024 SMA Europe congress, Roche presented a review of the SMAIS-ULM data in Part 2 of the SUNFISH trial.

Across all sub age groups the results showed that a larger proportion of those treated with risdiplam were likely to maintain or gain independence in their daily living activities than those on placebo:

Patient ages 12 – 25 years Maintained or Gained Independence in Activities of Daily Living
Treated with Risdiplam Placebo
Patient-reported outcome 69.8% 39.1%
Caregiver-reported outcome 66.4% 38.3%


17th March: Roche shared 3-year, longer-term data from the SUNFISH trial at the 2022 Muscular Dystrophy Association Virtual Clinical & Scientific Conference.


17th March: Roche shared new, 2-year, longer-term data from part 2 of the SUNFISH trial to be presented at the 2021 Muscular Dystrophy Association Virtual Clinical & Scientific Conference.


16th June: Roche presented two-year data on Part 1 of the SUNFISH trial, at the virtual Cure SMA Annual Conference.

6th February: Roche presented 1-year data from the pivotal Part 2 of their SUNFISH trial at the 2nd International Scientific and Clinical Congress on SMA in Evry, France.


12th November: Roche announced positive data from part 2 of the SUNFISH trial.

May: Roche presented positive data from its SUNFISH study at the 2019 American Academy of Neurology Annual Conference.


14th June: Roche announced all available screening slots in SUNFISH had been allocated by the active study centres, which meant that no new sites and countries would be activated for SUNFISH. This included the UK.


11th July: PTC Therapeutics presented an update on their SUNFISH trial of their splice-modifying drug, RG7916, at the 2017 Cure SMA Conference in Orlando, USA.


27th September: Roche have announced that they were to begin recruiting SMA patients to the SUNFISH trial.