Latest News: Treatments & Research

CHMP Publishes Positive Opinion on Risdiplam

26 February 2021 / Posted in: Treatments & Research

CHMP has recommended licensing approval of risdiplam for the treatment of 5q SMA in patients 2 months or older, with a clinical diagnosis of SMA Type 1, 2 or 3, or with 1-4 SMN2 copies. A final decision regarding approval is expected from the European Commission in the next two months, but could be as early as the end of March. Read more and what this means for the Early Access to Medicines Scheme (EAMS).

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Risdiplam and Salbumtamol - Clincians Answer a Question from Families

24 February 2021 / Posted in: Treatments & Research

Families have been asking if there is any advice about taking salbutamol at the same time as risdiplam.Our thanks to clinicians from the SMA REACH network who have provided an answer.

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NHS England's Advice to Clinicians about Offering Risdiplam EAMS

12 February 2021 / Posted in: Treatments & Research

This framework of advice is designed to assist clinicians to understand and interpret the risdiplam EAMS. Though we welcome this advice about who should have access to the risdiplam EAMS, we are very aware that many, particularly adults, are still unable to access nusinersen or risdiplam at their treatment centre. We continue to work hard to change this as a matter of urgency.

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NICE’s Public Consultation on Proposals for Changes to its Processes

08 February 2021 / Posted in: Information, Treatments & Research

This includes how NICE selects topics for its Highly Specialised Technologies programme which is one that has greatly concerned and affected the rare conditions community. The consultation is open until 15th April.

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Biogen: January Community Update on European / Global Access to Nusinersen

01 February 2021 / Posted in: Treatments & Research

Biogen has provided the latest update on access to nusinersen treatment around the world. There are now 29 European countries that have access to nusinersen via regular reimbursement, and this latest statement gives an updated situation in France.

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NICE’s Zolgensma Appraisal to go to a Second Committee Meeting

01 February 2021 / Posted in: Treatments & Research

NICE has announced that there will now be a second committee meeting on 10th February 2021. Registration to book to attend this virtual meeting is now open - closes 4pm on Friday 5th February.

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Risdiplam Early Access to Medicines Scheme (EAMS) - Clinicians Answer Further Questions

29 January 2021 / Posted in: Treatments & Research

Families and adults have been asking more questions about the clinical requirements for access to the EAMS. Our thanks to clinicians from the SMA REACH network who give answers, here.

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SMA UK's Submission to NICE Methods review

29 January 2021 / Posted in: Treatments & Research

NICE’s 6-week public consultation on the case for changing how it evaluates new medicines for use by the NHS started in November 2020. We attended a number of consultations and discussions run both by and for Patient Groups. You can read the key points we made in our December response to the consultation.

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Treatment Decisions in Newly Diagnosed Families with SMA

27 January 2021 / Posted in: Treatments & Research

A group of patient representatives from SMA Europe responded to European neuromuscular experts’ consensus statements on gene replacement therapy for SMA. Their short article, where they highlight some of the considerations that must be taken into account when considering treatment, was published in the European Journal of Paediatric Neurology.

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AMRC - Write to Boris Johnson to Save Charity-Funded Medical Research

11 January 2021 / Posted in: Information, Treatments & Research, Support

AMRC has reported on the devastating impact of covid on medical research charities who have had to make drastic cuts to their research funding. You can sign AMRC's letter calling for the Government to provide vital funding support to protect medical research and patients.

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