When will we know if the NHS will fund Risdiplam?
11 May 2021 / Posted in: Treatments & Research
Today, 3 patient experts will be speaking up for risdiplam, for all for whom it is clinically safe, at the first NICE committee meeting considering whether to recommend the treatment for NHS funding. We’ve outlined the stages and hoped-for timeline for when we’ll know the outcome.
New Guidelines To Improve Access To Nusinersen At Centres
10 May 2021 / Posted in: Treatments & Research
NHS England has just released guidelines about which children / adults should now be prioritised at centres actively providing nusinersen treatment, and what Centres should do if they’re having delays in starting treatment.
Survey Exploring Access to the Standards of Care for People who have SMA in the UK
10 May 2021 / Posted in: Information, Support
The John Walton Muscular Dystrophy Research Centre in Newcastle invites one respondent for each child / adult who has SMA to take part in gathering this important information that will help guide future practice.
Mental Health Awareness Week 2021
07 May 2021 / Posted in: Information, Support
This week (10th - 16th) is Mental Health Awareness Week and this year's theme is 'Nature'. Find out more about this, as well as how SMA UK can help with providing information and support.
Safety Signal for Zolgensma and Label Changes
05 May 2021 / Posted in: Treatments & Research
Novartis Gene Therapies have issued a community update which has been forwarded to all members of SMA Europe. All clinicians who will be discussing Zolgensma as a possible treatment option for a child will be aware of this update and be able to discuss what this means for them.
NICE Agrees to Access to Nusinersen for Those who have SMA Type 3 and Aren’t Able to Walk
04 May 2021 / Posted in: Treatments & Research
SMA UK is delighted with this decision which comes after almost two years of advocacy and hard work by families and adults - who have been impacted so distressingly by their exclusion from treatment - their clinicians, patient groups and Biogen. This decision also removes the rule which has meant that those who lose the ability to walk after 12 months of treatment or children who do not regain this ability, wouldn’t be eligible for it to continue.
Our Privacy Policy Has Been Updated
29 April 2021 / Posted in: Information
Following the UK’s exit from the E.U on the 1st January 2021, we have updated our Privacy Policy to reflect the changes to the GDPR (General Data Protection Act).
Webinar Recording - 'Zolgensma Treatment for SMA Type 1 in the UK'
29 April 2021 / Posted in: Treatments & Research
Our thanks to the leading health professionals who answered questions from the community and led this informative webinar, recorded 21st April 2021, about the treatment and what is going on to progress access. Watch the recording, read some of the key points, and see the answers we have so far to the many questions people have asked.
Spinal Muscular Atrophy UK Appoints New CEO
23 April 2021 / Posted in: Information
SMA UK is pleased to announce that Angela Smith-Morgan will be joining the charity as CEO from 26th April.
New Global Clinical Trial for Onasemnogene Abeparvovec (Zolgensma) Announced
23 April 2021 / Posted in: Treatments & Research
SMA UK welcomes a new global clinical trial that will be investigating the safety, tolerability and efficacy of intravenous Zolgensma in children who have SMA and weigh between 8.5 kgs and 21 kgs.
