Latest News: Treatments & Research
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.
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.
29 September 2020 / Posted in: Treatments & Research
Novartis has provided this update regarding their STRONG trial of intrathecal delivery of Zolgensma in older SMA patients.
28 September 2020 / Posted in: Treatments & Research
Our Scientific Research Correspondent, Dr. James Sleigh, has compiled this summary of the four trials of risdiplam: FIREFISH, SUNFISH, JEWELFISH and RAINBOWFISH.
25 September 2020 / Posted in: Treatments & Research
Based on the clinical and economic data from the manufacturer (Roche), NICE will be considering the clinical and cost effectiveness of risdiplam, and the views of patients and clinicians. We want to be sure to pass on your views to NICE when we make our Patient Group submission in October. Deadline: 11th October 2020.
Roche Answers Patient Group Questions about the UK Early Access to Medicines Scheme (EAMS) for Risdiplam
23 September 2020 / Posted in: Treatments & Research
This information is provided in response to a series of questions shared jointly with Roche in writing by UK patient organisations SMA UK, Muscular Dystrophy UK, and TreatSMA.
17 September 2020 / Posted in: Treatments & Research
Today, the Medicines and Healthcare Products Regulatory Agency (MRHA) announced access to Risdiplam for those who have SMA Type 1 or Type 2, aged 2 months and older, who are not suitable for authorised treatments.
14 September 2020 / Posted in: Treatments & Research
Biogen has provided the latest update on access to nusinersen treatment around the world. 29 European countries now have access to nusinersen via regular reimbursement.
14 September 2020 / Posted in: Information, Treatments & Research, Support
The first seminar of their 'MDUK Muscles Matter 2020' series took place during SMA Awareness Month and was an update on research into SMA and a panel discussion on potential treatments and managing the condition. Watch it here.