Latest News: Treatments & Research

Novartis Provided an Update on its Phase 1/2 Study in SMA Type 1

15 January 2020 / Posted in: Treatments & Research

Like Roche’s risdiplam (RG7916), branaplam (LMI070) is a small molecule drug that increases the amount of SMN protein made by the SMN2 gene. The study is progressing well, with a total of 29 infants receiving the study treatment.

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Scholar Rock Summarises Progress To Date With SRK-015

14 January 2020 / Posted in: Treatments & Research

SRK-015 is an experimental therapy directed at the muscle, that aims to reverse or restrict the atrophy and weakness experienced by SMA patients. Here, Scholar Rock summarise the latest progress.

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Roche Announces Global Compassionate Use Programme for Risdiplam

13 January 2020 / Posted in: Treatments & Research

This very positive development enables those who have SMA Type 1 global access to this promising treatment, with plans to expand access to those with SMA Type 2 later in the year. Roche UK has issued a statement with all the relevant information for UK patients.

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Do you or someone in your family have SMA Type 3? Are you / they currently not eligible for nusinersen treatment via the NHS?

10 January 2020 / Posted in: Treatments & Research

Complete our survey(s) by 16th February to let us and the other patient groups (MDUK and TreatSMA) know what impact this has had on you / your family.

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NICE Committee Meeting to Review the Possibility of Recommending that Access to the Treatment Zolgensma is Funded by the NHS Delayed

06 January 2020 / Posted in: Treatments & Research

NICE advised on 23rd Dec that the first committee discussion scheduled to take place on Thursday 26 March will not now take place. The evaluation will be rescheduled into the work programme and the updated timelines will be communicated, once known.

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SMA Europe Sets Out its Concerns about the AVXS-101 Zolgensma Global Compassionate Use Programme Announced in December 2019

06 January 2020 / Posted in: Treatments & Research

As members of SMA Europe, we endorse this statement made by SMA Europe on 26th December. It outlines the many concerns there are about this programme.

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Limited Global Access to Zolgensma announced by Avexis

20 December 2019 / Posted in: Treatments & Research

Clinicians and SMA patient groups in SMA Europe have welcomed the news that Avexis will be launching a global compassionate use programme in January 2020 for eligible children under 2 years of age, but have concerns about how it will operate.

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Clinicians & Patient Groups Requesting AveXis Clarification About Potential Limited Global Access to Zolgensma

19 December 2019 / Posted in: Treatments & Research

We're aware that complex and sensitive information regarding potential but very limited global access to the gene therapy treatment Zolgensma has been shared online. Clinicians and patient groups have expressed their concerns to the drug company AveXis requesting, as a matter of urgency, public clarification of the position.

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Nusinersen Managed Access Agreement – Update on Progress with Services for Adults

02 December 2019 / Posted in: Treatments & Research

NHS England has let the Patient Advocacy Groups (PAGs) know which hospitals now have contracts to offer adults treatment, and their plans to address any service gaps.

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FDA Grants Priority Review to Risdiplam for the Treatment of SMA

25 November 2019 / Posted in: Treatments & Research

The FDA’s decision will be announced on 24th May 2020. If approved, risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA.

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