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Leading health professionals talking directly to the Community about the latest developments. See the tabs below for previous updates:

January 2024: An Update on Zolgensma in the UK

In this latest webinar, we are joined by Consultant Paediatric Neurologists Dr Min Ong and Dr Imelda Hughes who give us an update on the Zolgensma programme in the UK and answer many of the questions being asked by the SMA Community. Amy Williams, who is mum to a little boy who has SMA Type 1, is the SMA UK host.

During this webinar, the terms ‘incident’, ‘pre-symptomatic’ and ‘prevalent’ are used. Please find definitions for these terms below:

  • Incident (all newly diagnosed)
  • Pre-symptomatic (newly diagnosed and not showing symptoms of SMA)
  • Prevalent (previously already diagnosed with SMA)

We’re very grateful to our panel of experts for their time in recording this webinar:

  • Fiona Marley – Head of Highly Specialised Commissioning, NHS England
  • Channa Hewamadduma – Consultant Neurologist at Sheffield
  • Sherryl Chatfield – Clinical Specialist Neuromuscular Physiotherapist at St George’s
  • Clare Galtrey – Consultant Neurologist at St George’s
  • Steven Jones – SMA UK host

This discussion covers, amongst other things: how adults who are thinking about accessing treatment can get a referral to a specialist neuromuscular centre; the possibility of offering adults Spinraza delivery through a port rather than lumbar puncture; the NICE review next year on whether to recommend NHS England continues funding treatments (and possible implications for Northern Ireland, Scotland and Wales); side effects some have been experiencing; clinical trials of other possible future treatments; updates on the Standards of Care.

This is our latest update on treatments for children webinar, recorded with a panel of consultant paediatric neurologists – Elizabeth Wraige (Evelina); Francesco Muntoni (GOSH); Anne-Marie Childs (Leeds) – and hosted by Amy Williams, mum of a little boy who has SMA Type 1. We’re hoping to release a webinar update on drug treatments for adults in the Autumn.

In this webinar, our three expert panellists, Elizabeth Wraige, Jennie Sheehan and Laurent Servais, discuss the UK Zolgensma programme and newborn screening for SMA with SMA UK Trustee and SMA Community member Toby Mildon.

Leading health experts discuss the progress of the roll-out of the nusinersen, risdiplam and Zolgensma treatment programmes with community member Mia Myers.

NHS England will now be funding this treatment. Leading health experts discuss the roll-out of the programme with community member Michaela Hollywood.

Sheffield’s Adults Neuromuscular Team talk through some useful information on baseline assessments and discussions for adults who have SMA who are making drug treatment decisions.