Latest News: Treatments & Research
SMA UK Sends Evidence to Biogen for the NICE Review Considering Whether People who have SMA Type 3 and Unable to Walk can Benefit from Nusinersen Treatment
23 November 2020 / Posted in: Treatments & Research
We are very aware that this review will be focusing on clinical evidence from Biogen and the clinical community. However, we consider it is also vital that NICE hears the wider ranging evidence we have gathered.
If you or your child live in England, have SMA Type 3 and have lost the ability to walk, please respond to this survey
20 November 2020 / Posted in: Treatments & Research
If NICE recommends that England's MAA for nusinersen should change to include you / your child, would you want to access this treatment? NHS England and the Neuromuscular Centres need to be ready for the possibility of a positive recommendation for change. SMA UK has agreed to put out this brief survey (closes 13th December) to help with any potential future planning. This does not guarantee a positive recommendation will be made.
Out of the Shadows - Report by The Neurological Alliance
12 November 2020 / Posted in: Information, Treatments & Research, Support
This new report, launched today, is calling for better treatment and care for the 150,000 children and adults living with a rare neurological condition, including SMA, in England.
SMA UK's and MDUK's Joint Submission to NICE as to Whether they Should Recommend that NHS England Funds Risdiplam
04 November 2020 / Posted in: Treatments & Research
Thank you to those of you who completed the survey we ran about your views on this - they were crucial for the submission we made. The first committee meeting is scheduled for 11th May 2021. You can see the submission and survey results through here.
I:DNA Immersive Art Installation
02 November 2020 / Posted in: Information, Treatments & Research, Support
Prof Felicity Boardman has done extensive research on the social and ethical implications of genomic medicine from the perspectives of people with genetic conditions. I:DNA is a virtual, immersive art installation which is an artistic representation of all this research. You can ask questions ahead of a live Q&A on 11th November.
NICE Announces Start of Review to Consider if People who have SMA Type 3 who're Unable to Walk can Benefit from Nusinersen Treatment
29 October 2020 / Posted in: Treatments & Research
NICE has today announced that it has begun the process to review data collected as part of the MAA for nusinersen. Specifically, it will consider whether people who have SMA Type 3 who are unable to walk can benefit from the treatment and therefore should be included in the MAA.
Scholar Rock Announces Encouraging Interim Data from the TOPAZ Phase 2 Trial of SRK-015
28 October 2020 / Posted in: Treatments & Research
SRK-015 is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by SMA patients. TOPAZ is evaluating SRK-015 across three cohorts of people, who have SMA Types 2 and 3, using motor function scales to measure meaningful endpoints.
Cell and Gene Therapy Engagement Workshops
23 October 2020 / Posted in: Information, Treatments & Research, Support
Genetic Alliance UK are organising a series of virtual workshops on the topic of cell and gene therapies, sometimes referred to as Advanced Therapy Medicinal Products (ATMPs). Date to register closes 26th October.
Independent Real-World Study Demonstrates Significant Improvements for Adults who have SMA Type 3, Treated with Nusinersen
06 October 2020 / Posted in: Treatments & Research
In response to a request from SMA Europe, Biogen has sent this community update which includes the observation that the most relevant treatment effect was seen in those with SMA Type 3 who were unable to walk without assistance at the start of treatment.
Roche: Update on Part 1 of the FIREFISH Risdiplam Clinical Trial
02 October 2020 / Posted in: Treatments & Research
Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of risdiplam in infants and determining the dose for Part 2. The data were presented at the virtual 25th International Annual Congress of the World Muscle Society.
