Latest News: Treatments & Research

Zolgensma Treatment for SMA Type 1 in the UK - Live Webinar with Experts

15 April 2021 / Posted in: Information, Treatments & Research, Support

On Wed 21st April at 5.30pm, this webinar with Prof. Francesco Muntoni (Consultant Paediatric Neurologist, GOSH), Dr Anne-Marie Childs (Consultant Paediatric Neurologist, Leeds) and Fiona Marley (Head of Highly Specialised Commissioning, NHS England), will be hosted by Martyn Sibley on the SMA UK Facebook and Twitter channels. You can send in your questions beforehand.

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SMA UK Responds to NICE’s Evaluation Consultation for Access to Zolgensma

13 April 2021 / Posted in: Treatments & Research

Last week, we supported NICE’s recommendations. We acknowledged that no current evidence or modelling was presented to demonstrate that Zolgensma could also provide health benefits for a proportion of children with SMA Type 2 and 3 and urged Novartis Gene Therapies to continue to work to address this gap in access to the treatment.

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Scholar Rock Announces Results from TOPAZ Phase 2 Trial in SMA Types 2 and 3

08 April 2021 / Posted in: Treatments & Research

Apitegromab (also known as SRK-015) aims to reverse or restrict the muscle atrophy and weakness experienced by people living with SMA. Topaz is a clinical trial designed to assess the safety and efficacy of apitegromab in people living with SMA Types 2 and Type 3. The 12-month data announced yesterday by Scholar Rock demonstrate early therapeutic potential.

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Results of SMA Europe’s 2nd Pan-European Survey on Patient Expectations Published

30 March 2021 / Posted in: Information, Treatments & Research

Completed by 1,474 people from 26 European countries, this study confirms what we have been saying in the UK in particular that stabilisation of someone’s SMA and even minor changes in their ability have meaningful impacts on daily life. It’s now summarised on the SMA Europe website with a link to the full publication.

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Risdiplam Receives EU Marketing Authorisation

30 March 2021 / Posted in: Treatments & Research

Risdiplam has now received marketing authorisation in the European Union (EU) for the treatment of 5q SMA in those aged 2 months and older, with a clinical diagnosis of Type 1, Type 2 or Type 3 or with one to four SMN2 copies - this will apply to Northern Ireland (NI) and impact on the NI EAMS. Risdiplam remains an unlicensed medicine in the rest of the UK, with Roche anticipating marketing authorisation in May 2021.

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Launch of the European Newborn Screening Alliance Whitepaper on the need for NBS Programmes to include SMA

26 March 2021 / Posted in: Information, Treatments & Research

Today, Alliance leaders, MEPs and others, spoke passionately of why action for NBS is needed across Europe now and you can watch a recording of the event. The whitepaper covers the evidence and arguments; this and other information is here. The UK NBS Alliance (of which we are a member) is meeting at the end of this month and we'll update you with its activity as soon as possible.

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Roche Presents New Two-Year Data From SUNFISH (Part 2) Trial

17 March 2021 / Posted in: Treatments & Research

New two-year data show Roche’s Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 years, who have SMA Type 2 or Type 3.

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Update on Access to Zolgensma Treatment in England

16 March 2021 / Posted in: Treatments & Research

Following the announcements on Monday 8 March 2021, we wanted to clarify how the NHS England announcement relates to the NICE draft recommendations, especially how they potentially enable different groups of children to access Zolgensma. We have therefore been in touch with NHS England asking for an update and to clarify as much as is possible at this stage.

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Update on progress of the NICE Type 3 evidence review

15 March 2021 / Posted in: Treatments & Research

The nusinersen clinical eligibility criteria review is progressing as planned to the anticipated timeline. Patient groups and clinicians met in March 2021 with NICE. The NICE website now states that publication of the final guidance is expected in June 2021.

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SMA PhD Partnership - SMA UK and MDUK

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

SMA UK and MDUK are delighted to announce our SMA PhD Partnership! Applications are now open for one 4-year PhD studentship in 5q SMA research as part of Muscular Dystrophy UK’s Grant Round 2021, and close at 5pm on Tuesday 1st June 2021.

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