Roche to Start MANATEE, a Drug Combination Study, in Early 2022
22 October 2021 / Posted in: Treatments & Research
Roche will initiate MANATEE, a new global Phase 2/3 clinical study to evaluate the safety and efficacy of GYM329 (RO7204239), an investigational anti-myostatin antibody targeting muscle growth, in combination with risdiplam in SMA.
New Survey: Being a Teenager With SMA
21 October 2021 / Posted in: Information, Support
Are you aged 13-18? Do you have a diagnosis of SMA? Jordanne has SMA Type 2 and is currently studying a master's degree in clinicial psychology. She's interested to hear more about how SMA affects the emotions and quality of life of people aged 13-18 years.
Online Event: Newborn Screening: "What Should Be Screened For and How?"
14 October 2021 / Posted in: Information, Treatments & Research, Support
Recently, patient groups and others have asked for conditions, including SMA, to be added to the nine that are currently screened for in newborns, but this has not been done. This event, on 3rd Nov, will see the Director of Programmes for the UK National Screening Committee, and other experts and advocates, discuss what should be screened for in newborns and how.
Survey For Young People with Neuromuscular Conditions About Fatigue
11 October 2021 / Posted in: Information
A trainee clinical psychologist at the University of Bath is running this survey about fatigue for young people (age 10-24 years) who have neuromuscular conditions. The survey takes approximately 20-30 minutes to complete and there is also an optional prize draw for participants to enter.
New Research Project for Adults who have SMA and Parents / Caregivers of those aged 2-18 years
11 October 2021 / Posted in: Information, Support
Liberating Research is looking to speak to people with SMA and caregivers / parents of people living with SMA, to understand better the everyday life experiences and needs of people affected by the condition. Everyone who participates will be paid £80 as a token of appreciation for their time. Closes 4th November.
We're Hiring! New Advocacy Lead Job Role
06 October 2021 / Posted in: Information
If you’re looking to make a major difference to the lives of adults and families living with SMA, this high-profile Advocacy Lead role that we’ve created could be the new challenge you’ve been waiting for. Applications close 1st November.
SMA UK and MDUK’s Submission on Risdiplam now with the Scottish Medicines Consortium
04 October 2021 / Posted in: Treatments & Research
Our thanks to all of you who responded to our surveys with your views and for other comments we have received. They were invaluable and we hope we have done them justice. We will keep you posted when we hear more about the next stages.
NMDT update
24 September 2021 / Posted in: Treatments & Research
Thank you to the NMDT for letting us know they have now invited Centres to refer children in England who have SMA Type 1 and who weigh less than 13.5kgs for assessment for Zolgensma treatment.
Mindfulness Mondays
23 September 2021 / Posted in: Information, Support
SMA UK are launching a new 6-week Mindfulness Monday programme for those in the SMA Community aged 18+. Starting on the 4th October, these live, drop-in style group sessions of 30 minutes every Monday lunchtime will be led by occupational therapist Josephine Dolan-Dufourd. Register for free here!
MDUK Muscles Matter 2021 SMA (Paediatric Focus) Seminar: Watch Online
22 September 2021 / Posted in: Information, Support
On 23rd August, we were delighted to be on the panel presenting at Muscular DystrophyUK's Muscles Matter 21 Online Seminar. The session had a paediatric SMA focus, also featuring a panel of paediatric neurologists and physiotherapists, and is now available to watch online.
