2024

14th October: Majority of children in RAINBOWFISH Trial with pre-symptomatic SMA who began risdiplam treatment before six weeks of age can sit, stand and walk independently

2023

23rd February: An interim analysis of data from the JEWELFISH Trial was published in the scientific journal, Neurology and Therapy.

2022

17th March: The latest risdiplam (Evrysdi) data updates were presented virtually at the 2022 MDA Clinical and Scientific Conference on 13th – 16th March. These were for the SUNFISH and RAINBOWFISH trials.

To date, more than 5,000 people had been treated with Evrysdi in clinical trials, compassionate use or real-world settings. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

2021

14th June: Preliminary results from the RAINBOWFISH and JEWELFISH trials were presented at the Cure SMA 2021 Virtual SMA Research & Clinical Care Meeting, which took place from 9th to 11th June 2021.

19th April: Roche presented new two-year data from part 2 of its FIREFISH study at the 73rd American Academy of Neurology (ANN) Annual Meeting held virtually 17th – 22nd April 2021.

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

February: Data from part 1 of FIREFISH, a dose-determining study, were published in the New England Journal of Medicine.

2020

October: Data on Part 1 of the FIREFISH trial presented at the virtual 25th Annual Congress of the World Muscle Society showed that infants treated with the therapeutic dose of risdiplam continued to improve and achieve motor milestones.

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.

2019

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

May: At the 2019 American Academy of Neurology Annual Conference, Roche presented positive data from its SUNFISH study and reported that enrollment for the FIREFISH study was now complete with preliminary results expected in early 2020.

2018

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

June: PTC Therapeutics presented preliminary data at the 2018 Cure SMA conference in Dallas indicating that risdiplam appears to be well tolerated at all doses tested and to date has not caused any drug-related adverse events causing patient withdrawal from the trial.

2017

August: Roche had begun to enrol SMA patients in the FIREFISH Phase II trials of their splice-modifying drug, RG7916 (also known as RO7034067) in the US, Belgium, Italy, Switzerland, and Turkey

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.

2016

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