05 May 2021 / Posted in: Treatments & Research
Novartis Gene Therapies have issued a community update which has been forwarded to all members of SMA Europe. All clinicians who will be discussing Zolgensma as a possible treatment option for a child will be aware of this update and be able to discuss what this means for them.
04 May 2021 / Posted in: Treatments & Research
SMA UK is delighted with this decision which comes after almost two years of advocacy and hard work by families and adults - who have been impacted so distressingly by their exclusion from treatment - their clinicians, patient groups and Biogen. This decision also removes the rule which has meant that those who lose the ability to walk after 12 months of treatment or children who do not regain this ability, wouldn’t be eligible for it to continue.
29 April 2021 / Posted in: Information
29 April 2021 / Posted in: Treatments & Research
Our thanks to the leading health professionals who answered questions from the community and led this informative webinar, recorded 21st April 2021, about the treatment and what is going on to progress access. Watch the recording, read some of the key points, and see the answers we have so far to the many questions people have asked.
23 April 2021 / Posted in: Information
SMA UK is pleased to announce that Angela Smith-Morgan will be joining the charity as CEO from 26th April.
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.