Survey for All Adults who have 5q SMA (Types 1, 2 or 3) and Live in the UK
18 February 2020 / Posted in: Treatments & Research
To help with planning the roll-out of the nusinersen treatment programme, we want to hear from all adults who live in the UK and who have 5q SMA (Types 1, 2 or 3) to find out who does and who doesn’t want to access nusinersen (Spinraza) treatment. Survey closes 15th March.
Walking the Great North Run for SMA UK
14 February 2020 / Posted in: Help Us
Dot and Pat decided to walk the Great North Run last year
Changing Places: Funding For Hospitals
07 February 2020 / Posted in: Information, Support
As of January 2020, £500,000 of the £2 million fund provided by the Department of Health and Social Care has now been allocated for 16 new Changing Places toilets being installed at hospitals. The rest of the fund is open to new applications from NHS Hospital Trusts in England.
User Interviews re: the Redevelopment of the UK National Screening Committee Website
07 February 2020 / Posted in: Information, Support
Public Health England (PHE) Digital is conducting this research and is seeking volunteers to provide feedback. Those who take part will receive a £50 voucher to thank them for their time.
Roche’s Risdiplam Showed Significant Improvement in Motor Function in People who have SMA Type 2 or 3
06 February 2020 / Posted in: Treatments & Research
Their SUNFISH trial showed change from the baseline in the primary endpoint of the Motor Function Measure scale was significantly greater in people treated with risdiplam, compared to the placebo. The Revised Upper Limb Module also showed an improvement.
The Great North Run for Abigail
30 January 2020 / Posted in: Help Us
Hazel is running the Great North Run this year in memory of Abigail.
Roche’s Risdiplam Meets Primary Endpoint in pivotal FIREFISH Trial in Infants who have SMA Type 1
23 January 2020 / Posted in: Treatments & Research
Roche have put out a press release showing positive endpoint results from their FIREFISH study of risdiplam with infants aged 1-7 months who have SMA Type 1.
New Survey Launched to Understand Activity Needs of Disabled Children
16 January 2020 / Posted in: Information, Support
The ukactive Research Institute has announced a three-year research project with national disability charity, Sense, to understand better the needs of parents and families of disabled children. Survey closes 15th March.
Novartis Provided an Update on its Phase 1/2 Study in SMA Type 1
15 January 2020 / Posted in: Treatments & Research
Like Roche’s risdiplam (RG7916), branaplam (LMI070) is a small molecule drug that increases the amount of SMN protein made by the SMN2 gene. The study is progressing well, with a total of 29 infants receiving the study treatment.
Scholar Rock Summarises Progress To Date With SRK-015
14 January 2020 / Posted in: Treatments & Research
SRK-015 is an experimental therapy directed at the muscle, that aims to reverse or restrict the atrophy and weakness experienced by SMA patients. Here, Scholar Rock summarise the latest progress.
