Latest News: Treatments & Research
Biogen to Start New Trial to Evaluate Higher Doses of Nusinersen in Later-Onset SMA
17 September 2021 / Posted in: Treatments & Research
Biogen plans to initiate a new clinical trial called ASCEND, which will evaluate whether treatment with a higher dose of nusinersen has the potential to improve clinical outcomes and address unmet medical needs in people with later-onset SMA who were previously treated with risdiplam.
Access to Risdiplam in Scotland: Your Views
02 September 2021 / Posted in: Treatments & Research
The SMC has started its review of whether to recommend risdiplam for funding by the NHS in Scotland. SMA UK and MDUK are wanting our joint submission to reflect the views of people in Scotland who are affected by SMA, including those who are bereaved by SMA, so we’re putting out this short survey with a closing date of Thursday 16th September.
MDUK Muscles Matter 2021 SMA (Paediatric Focus) Seminar
19 August 2021 / Posted in: Information, Treatments & Research
Muscular Dystrophy UK are holding this online seminar on Monday 23rd August, 10am-12pm, to keep you up-to-date with the latest research into SMA. You will have the chance to hear from experts about progress into research and treatments, and get the opportunity to ask questions.
NICE methods, Process and Topic Selection for Health Technology Evaluation: Proposals for Change Consultation Now Open
19 August 2021 / Posted in: Information, Treatments & Research
This eight-week consultation on the final stage of NICE’s methods and processes review is now open for any responses by 13th October. We will attend meetings with partners and alliances to review what is being proposed and join with responses.
Phase 3 Trial to Evaluate OAV-101 Intrathecal (IT) in People with SMA
17 August 2021 / Posted in: Treatments & Research
The FDA in the US has removed the partial clinical trial hold initiated in October 2019 and determined that Novartis Gene Therapies’ investigational compound OAV-101 IT clinical trial for people with SMA may proceed. Novartis now plans to evaluate the clinical efficacy, safety and tolerability of OAV-101 IT in treatment naïve people with SMA who are between two and 18 years of age, able to sit, but have never walked.
Scotland Starts to Progress Review of Whether to Recommend Risdiplam for NHS Funding
17 August 2021 / Posted in: Treatments & Research
We are pleased to see that the Scottish Medicines Consortium has now made this announcement. We and the other patient groups will be making submissions in time for the deadline for these of 4th October 2021.
Appraisal of risdiplam in Scotland delayed further
03 August 2021 / Posted in: Treatments & Research
SMA UK, MDUK and TreatSMA have today written to the Scottish Medicines Consortium (SMC) to raise their concerns about the further delays with processing Roche’s submission for risdiplam.
Data for Evrysdi Published in NEJM shows Significant Improvement in Survival and Motor Milestones in Babies with SMA Type 1
30 July 2021 / Posted in: Treatments & Research
The study, evaluating the efficacy and safety of Evrysdi™ (risdiplam) in babies aged 1-7 months old with symptomatic SMA Type 1, met its primary endpoint with 29% of infants sitting without support for at least five seconds by month 12 - a milestone not seen in the natural course of the condition.
Community Update: Novartis Discontinues Development of Branaplam
21 July 2021 / Posted in: Treatments & Research
Novartis have confirmed that they've made the difficult decision to discontinue the development of branaplam (also known as LMI070) for the treatment of SMA. They explain that their decision was "made as the result of rapid advancements in the SMA treatment landscape in recent years."
JCVI Issues Advice on COVID-19 Vaccination of Children and Young People
20 July 2021 / Posted in: Information, Treatments & Research, Support
The JCVI has looked at the available evidence around vaccinating children and young people under the age of 18. They have now advised that children at increased risk of serious COVID-19 disease are offered the Pfizer-BioNTech vaccine. That includes children aged 12 to 15 with severe neurodisabilities, Down’s syndrome, immunosuppression and multiple or severe learning disabilities.
