Early Access to Medicines Scheme (EAMS) announced for Risdiplam in the UK
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.
Left In Lockdown - DCP Survey Results
15 September 2020 / Posted in: Information, Support
Back in May, the Disabled Children's Partnership ran a survey to understand more about the impact the coronavirus pandemic and lockdown was having on disabled children and young people, their siblings and parents. Over 4,000 people completed the survey. Read about the results.
Biogen: September Community Update on European / Global Access to Nusinersen
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.
Muscular Dystrophy UK - SMA Seminar
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.
AveXis Renamed Novartis Gene Therapies
03 September 2020 / Posted in: Treatments & Research
Novartis has renamed the previously acquired AveXis - the pharmaceutical company behind the gene therapy treatment, Zolgensma - to Novartis Gene Therapies. The change signifies the growing importance of gene therapy.
Same But Different - 'A Glimmer of Hope' Calendar Photography Competition
03 September 2020 / Posted in: Information, Support
The winning images will be used to create a calendar that will celebrate the people behind rare conditions, as well as helping to raise awareness. The competition is open now and runs until the 22nd September.
New Extensions to the Access To Work Scheme Announced
02 September 2020 / Posted in: Information, Support
Covid-19 is changing the way we work and impacting working environments and arrangements. With many employers asking workers to adapt to a new environment, Access to Work can play a key role in supporting disabled people to retain, return to and move into employment.
SMA Europe and Partners Launch the European Alliance for Newborn Screening
31 August 2020 / Posted in: Treatments & Research
Early diagnosis and treatment of SMA leads to better outcomes. Launched today, on the last day of SMA Awareness Month, this Alliance brings together all stakeholders (EAMDA, EURORDIS, TREAT-NMD, AveXis, Biogen, and Roche) with the aim, by 2025, for newborn screening programmes in Europe to include a test for SMA.
Contact - Back to School Advice (England)
28 August 2020 / Posted in: Information, Support
The charity Contact for Families with Disabled Children, have published some guidance around this to help answer questions and concerns that parents may have ahead of their children going back to school in September.
Survey Exploring Access to the Standards of Care for People who have SMA in the UK
28 August 2020 / 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.
