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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Have your say on where to install Changing Places Toilets in England

09 April 2021 / Posted in: Information, Support

If you use Changing Places toilets, or need to use them, you can add your voice to the UK’s biggest consultation about these vital facilities. By doing the 10-minute survey, you’ll help inform where, and in which buildings, these facilities are needed the most in England. Survey closes: 23rd April.

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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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DCP Campaign - Call for a Recovery Plan

07 April 2021 / Posted in: Information, Support

Join this campaign from the Disabled Children's Partnership by emailing the Government to share your experiences and call on them for a Covid19 recovery plan for disabled children, young people and families.

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Living With SMA Podcast - New Episode Available!

07 April 2021 / Posted in: Information, Support

The next episode of our Living With SMA podcast is available to listen to now! In 'My Experience of Home Adaptations', SMA UK Host Josh Wintersgill talks to Julian Fiorentini, who shares his personal journey from school days, through to living in a care home and his experience of making adaptations to his home, as an adult.

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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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Easter Bank Holiday

26 March 2021 / Posted in: Information, Support

Our office will be closed from 3.30pm Thursday 1st April and will re-open from 9am on Tuesday 6th April. Find out more about where to find information and support during this time.

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