Latest News: Treatments & Research

Thank You

09 March 2021 / Posted in: Information, Treatments & Research, Support

Following yesterday's announcements, SMA UK would like to thank all those involved.

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NICE Releases its Evaluation Consultation Document (ECD) on Zolgensma in England

08 March 2021 / Posted in: Treatments & Research

NICE has backed NHS England’s announcement this morning to fast-track the introduction of this innovative gene therapy so that it becomes available in England before NICE publishes its final guidance. So that NICE can complete its appraisal of the treatment, it’s ECD is now available on their website and is open for anyone to comment by the closing date of 5pm Tuesday 6th April 2021.

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SMA Gene Therapy Treatment Zolgensma Accepted for Restricted Use within NHS Scotland

08 March 2021 / Posted in: Treatments & Research

The Scottish Medicines Consortium has just announced the positive news that it has accepted Zolgensma for restricted use for SMA Type 1 within NHS Scotland. This follows NHS England’s and NICE’s announcements earlier today. Read further details, including about eligibility.

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NHS England Announces Access to Zolgensma for Eligible Children who have SMA Type 1

08 March 2021 / Posted in: Treatments & Research

NHS England’s announcement of this landmark agreement is positive news for parents of some children who have SMA Type 1 who may now be eligible for access to this one-time gene therapy.

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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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