23 April 2021 / Posted in: Treatments & Research
SMA UK welcomes a new global clinical trial that will be investigating the safety, tolerability and efficacy of intravenous Zolgensma in children who have SMA and weigh between 8.5 kgs and 21 kgs.
22 April 2021 / Posted in: Treatments & Research
Biogen presents new data from its DEVOTE study of Spinraza (nusinersen) as well as an update on biomarkers and digital tools to enhance disease monitoring, at the 73rd American Academy of Neurology (AAN) Annual Meeting.
22 April 2021 / Posted in: Information, Support
Sense have launched this campaign, which is about taking action to tackle loneliness among disabled people and making society more inclusive. The campaign is calling for more investment in services to tackle loneliness and offer mental health support for disabled people. Sign their pledge and play your part in making society more inclusive.
21 April 2021 / Posted in: Information, Support
BBC News is carrying out a survey to understand how Covid-19 has affected deaf and disabled people. They are trying to reach as many people as possible to get the biggest, most detailed picture they can.
19 April 2021 / Posted in: Treatments & Research
Roche has shared new two-year data from part 2 of its FIREFISH study. FIREFISH is assessing Evrysdi™ (risdiplam) in the treatment of infants aged 1-7 months at enrollment, with symptomatic SMA Type 1. The data build upon one-year findings from FIREFISH Part 2, which measures the ability to sit without support for at least five seconds as its primary endpoint.
15 April 2021 / Posted in: Information, Treatments & Research, Support
On Wed 21st April at 5.30pm, this webinar with Prof. Francesco Muntoni (Consultant Paediatric Neurologist, GOSH), Dr Anne-Marie Childs (Consultant Paediatric Neurologist, Leeds) and Fiona Marley (Head of Highly Specialised Commissioning, NHS England), will be hosted by Martyn Sibley on the SMA UK Facebook and Twitter channels. You can send in your questions beforehand.
13 April 2021 / Posted in: Treatments & Research
Last week, we supported NICE’s recommendations. We acknowledged that no current evidence or modelling was presented to demonstrate that Zolgensma could also provide health benefits for a proportion of children with SMA Type 2 and 3 and urged Novartis Gene Therapies to continue to work to address this gap in access to the treatment.
09 April 2021 / Posted in: Information, Support
If you use Changing Places toilets, or need to use them, you can add your voice to the UK’s biggest consultation about these vital facilities. By doing the 10-minute survey, you’ll help inform where, and in which buildings, these facilities are needed the most in England. Survey closes: 23rd April.
08 April 2021 / Posted in: Treatments & Research
Apitegromab (also known as SRK-015) aims to reverse or restrict the muscle atrophy and weakness experienced by people living with SMA. Topaz is a clinical trial designed to assess the safety and efficacy of apitegromab in people living with SMA Types 2 and Type 3. The 12-month data announced yesterday by Scholar Rock demonstrate early therapeutic potential.
07 April 2021 / Posted in: Information, Support
Join this campaign from the Disabled Children's Partnership by emailing the Government to share your experiences and call on them for a Covid19 recovery plan for disabled children, young people and families.