Biogen to Initiate Phase 4 Study Evaluating Benefit of Spinraza® in Patients Treated With Zolgensma®
21 July 2020 / Posted in: Treatments & Research
Biogen have announced today that it plans to initiate this clinical study, called RESPOND, to examine the clinical benefit, and assess the safety, of Spinraza in infants and children who have SMA, who still have unmet clinical needs following treatment with the gene therapy treatment Zolgensma.
Changing Places Compulsory for New Buildings in England from 2021
20 July 2020 / Posted in: Information, Support
Shopping centres, supermarkets, sports and arts venues will be required to include at least one Changing Place. There was also further funding announced for more Changing Places toilets at service stations in England.
Face Masks for Children who have a Rare Condition
20 July 2020 / Posted in: Information, Support
The not-for-profit arts organisation Same But Different are giving these away free for children in the UK who have a rare condition.
Impact of COVID-19 on Physical Activity and Wellbeing in Adults who have a Neurologically-Related Mobility Disability
16 July 2020 / Posted in: Information, Support
The University of Birmingham is running this survey to find about more about the impact that the pandemic has had on physical activity and health-related quality of life.
Our Thanks to our Retiring Trustees
16 July 2020 / Posted in: Information
Our July board meeting saw a number of changes to the composition of our Board of Trustees, including 3 members stepping down - we'd like to thank them for their tireless and dedicated support, their advocacy for our cause, as well as their wisdom and counsel.
NICE to discuss Zolgensma at 8th October Committee meeting
14 July 2020 / Posted in: Treatments & Research
Onasemnogene abeparvovec (Zolgensma) has been rescheduled into the National Institute for Health and Care Excellence’s (NICE’s) work programme and is due to be discussed at committee on this date
European consensus statement concerning treatment with Zolgensma
13 July 2020 / Posted in: Treatments & Research
A group of prominent European neuromuscular experts have provided eleven consensus statements covering many aspects of treatment with onasemnogene abeparvovec (Zolgensma).
Effect of risdiplam on Female Fertility and Menstruation
10 July 2020 / Posted in: Treatments & Research
Roche has responded to the question we asked on behalf of the Community about this.
Further Small Studies Support the Use of Nusinersen for SMA Treatment in Adults
03 July 2020 / Posted in: Treatments & Research
Three scientific reports have been published that present observations from adults who have SMA treated with repeated injections of nusinersen into the fluid bathing the spinal cord and brain.
Over 150 Organisations Call on Government to Prioritise Children as Much as Health and the Economy
30 June 2020 / Posted in: Information, Support
The impact of the COVID-19 pandemic has been widespread, affecting everybody in the country, and, in particular, this generation of children face unprecedented threats to their childhoods and life chances. They deserve an unprecedented response.
