Latest News: Treatments & Research

NICE to discuss Zolgensma at 8th October Committee meeting

14 July 2020 / Posted in: Treatments & Research

Onasemnogene abeparvovec (Zolgensma) has been rescheduled into the National Institute for Health and Care Excellence’s (NICE’s) work programme and is due to be discussed at committee on this date

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European consensus statement concerning treatment with Zolgensma

13 July 2020 / Posted in: Treatments & Research

A group of prominent European neuromuscular experts have provided eleven consensus statements covering many aspects of treatment with onasemnogene abeparvovec (Zolgensma).

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Effect of risdiplam on Female Fertility and Menstruation

10 July 2020 / Posted in: Treatments & Research

Roche has responded to the question we asked on behalf of the Community about this.

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Further Small Studies Support the Use of Nusinersen for SMA Treatment in Adults

03 July 2020 / Posted in: Treatments & Research

Three scientific reports have been published that present observations from adults who have SMA treated with repeated injections of nusinersen into the fluid bathing the spinal cord and brain.

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Progress with Access to Nusinersen in European Countries for Adults who have SMA

25 June 2020 / Posted in: Treatments & Research

Thank you to the SMA Europe member countries who responded to our request for information about the reality of access to treatment by adults in their country. Some provided other information about access in general, and testimonials.

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Roche Presents New Data on SUNFISH & JEWELFISH Trials at the Cure SMA Annual 2020 virtual Conference

16 June 2020 / Posted in: Treatments & Research

Roche presented this new data at the virtual Cure SMA Annual Conference, which took place between 8th and 12th June.

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Further Positive Results in NURTURE Study with Pre-Symptomatic SMA Patients Treated with Nusinersen

12 June 2020 / Posted in: Treatments & Research

New results from the NURTURE study show that pre-symptomatic SMA patients treated with nusinersen continue to demonstrate sustained benefit from treatment.

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NICE / NHS England Answer the Patient Groups FAQS about Adults’ Access to Nusinersen Treatment in England

11 June 2020 / Posted in: Treatments & Research

NHS England and NICE have provided further responses to these FAQs, around adults' access to nusinersen, raised in August 2019 by the patient groups.

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Information Statement from the NHS England Clinical Panel for Nusinersen

11 June 2020 / Posted in: Treatments & Research

This is about access to the risdiplam Compassionate Use Programme for anyone who has SMA Type 1 or 2 who is already receiving nusinersen but who has had their treatment interrupted because of COVID-19.

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NICE & NHS England Answer Further FAQS about the Nusinersen MAA; Biogen Gives Update on Global Access

09 June 2020 / Posted in: Treatments & Research

You can read these communications in this update. Answers to our FAQs about adult services will also be coming soon.

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