Latest News: Treatments & Research

Cell and Gene Therapy Engagement Workshops

23 October 2020 / Posted in: Information, Treatments & Research, Support

Genetic Alliance UK are organising a series of virtual workshops on the topic of cell and gene therapies, sometimes referred to as Advanced Therapy Medicinal Products (ATMPs). Date to register closes 26th October.

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Independent Real-World Study Demonstrates Significant Improvements for Adults who have SMA Type 3, Treated with Nusinersen

06 October 2020 / Posted in: Treatments & Research

In response to a request from SMA Europe, Biogen has sent this community update which includes the observation that the most relevant treatment effect was seen in those with SMA Type 3 who were unable to walk without assistance at the start of treatment.

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Roche: Update on Part 1 of the FIREFISH Risdiplam Clinical Trial

02 October 2020 / Posted in: Treatments & Research

Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of risdiplam in infants and determining the dose for Part 2. The data were presented at the virtual 25th International Annual Congress of the World Muscle Society.

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Community Update on AVXS-101 Intrathecal Clinical Development Program

29 September 2020 / Posted in: Treatments & Research

Novartis has provided this update regarding their STRONG trial of intrathecal delivery of Zolgensma in older SMA patients.

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Summary of the Outcomes of the Clinical Trials of Risdiplam: September 2020

28 September 2020 / Posted in: Treatments & Research

Our Scientific Research Correspondent, Dr. James Sleigh, has compiled this summary of the four trials of risdiplam: FIREFISH, SUNFISH, JEWELFISH and RAINBOWFISH.

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Should NICE recommend that the NHS funds risdiplam and, if so, who for? Let us know your views

25 September 2020 / Posted in: Treatments & Research

Based on the clinical and economic data from the manufacturer (Roche), NICE will be considering the clinical and cost effectiveness of risdiplam, and the views of patients and clinicians. We want to be sure to pass on your views to NICE when we make our Patient Group submission in October. Deadline: 11th October 2020.

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Roche Answers Patient Group Questions about the UK Early Access to Medicines Scheme (EAMS) for Risdiplam

23 September 2020 / Posted in: Treatments & Research

This information is provided in response to a series of questions shared jointly with Roche in writing by UK patient organisations SMA UK, Muscular Dystrophy UK, and TreatSMA.

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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.

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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.

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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.

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