Latest News: Treatments & Research

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.

Read full story

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.

Read full story

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.

Read full story

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.

Read full story

First patient treated in Biogen’s global RESPOND study of nusinersen, following treatment with Zolgensma™

11 January 2021 / Posted in: Treatments & Research

The Phase 4 open-label, multicentre study will examine the clinical benefit and safety of nusinersen treatment in infants and children with SMA who have unmet clinical needs following previous treatment with Zolgensma.

Read full story

New UK Rare Diseases Framework Published January 2021

10 January 2021 / Posted in: Information, Treatments & Research

The Framework demonstrates the commitment of UK governments to improve the lives of people with rare conditions, and address key challenges, over the next five years.

Read full story

Information Statement about how Access to Nusinersen Treatment is Affected by COVID-19

08 January 2021 / Posted in: Treatments & Research

In April 2020, advice from clinical experts and discussions with the Patient Groups informed a statement from NICE, NHS England and NHS Improvement. NICE have reviewed this and confirm that the flexibility and guidance in this statement remains in place.

Read full story

Children with SMA and Covid 19 - Spanish Study

06 January 2021 / Posted in: Treatments & Research

A study by the Spanish Society for Pediatric Neurology of a small number of children with SMA who caught COVID-19 concludes that, "The course of COVID-19 in children with neuromuscular disorders may not be as severe as expected…". The authors do though advise that their study, "has several limitations worth noting”.

Read full story

Cambridge University Hospitals NHS Foundation Trust Joins the Risdiplam EAMS for Adults

23 December 2020 / Posted in: Treatments & Research

We are pleased to have received notice that the Trust’s Joint Drugs and Therapeutics Committee (JDTC) has now made this decision, joining more than 15 other Trusts prescribing risdiplam via the Early Access to Medicines Scheme. Adults, clinicians and patient groups worked hard to provide information to enable the Committee to reach this position.

Read full story

Roche - End of Year Summary

21 December 2020 / Posted in: Treatments & Research

Roche has provided SMA Europe with a summary of their global activities carried out this year which may be of interest. This includes information about the risdiplam clinical development programme and progress towards their goal of making risdiplam available to all patients who may benefit from the treatment.

Read full story