Respond to PEEPs (Personal Emergency Evacuation Plans) Consultation

23 June 2021 / Posted in: Information, Support

The Government is running a consultation seeking views on new proposals to implement the Grenfell Tower Inquiry Phase 1 recommendations on 'PEEPs' in high-rise residential buildings. Claddag - a Leaseholder Disability Action Group - have put together some suggested responses to help if you'd like to respond to the consultation which closes on 19th July.

Read full story

Save the date: SMA Europe - 3rd International Scientific and Clinical Congress on SMA

21 June 2021 / Posted in: Information

SMA Europe has announced its third International Scientific and Clinical Congress on Spinal Muscular Atrophy, which will take place between 9th-11th February 2022 at the International Convention Centre (CCIB) in Barcelona, Spain.

Read full story

Contact: Write to the JVCI about Vaccines for Clinically Vulnerable Children

17 June 2021 / Posted in: Information, Support

National charity Contact have prepared a template letter for parents / carers who want the Joint Committee on Vaccination and Immunisation (JCVI) to recommend offering a Covid-19 vaccine as soon as possible to children with underlying health conditions / who are clinically vulnerable.

Read full story

Contact Survey: Counting the Costs 2021

15 June 2021 / Posted in: Information, Support

Contact is running this new survey to find out how the pandemic has affected your family finances and how you're feeling about your future family finances. It takes about 25 minutes to complete and there is an opportunity at the end to win one of three £100 vouchers. The deadline to complete is Wednesday 7 July at 9am.

Read full story

Evrysdi™ Improves Motor Function in Pre-Symptomatic Babies

14 June 2021 / Posted in: Treatments & Research

In response to a request from SMA Europe, Roche has shared new interim data from two studies evaluating risdiplam in people with SMA. The trials included pre-symptomatic babies (RAINBOWFISH) or patients who have been previously treated with other SMA-targeting therapies (JEWELFISH). The findings were presented at the Cure SMA 2021 Virtual SMA Research & Clinical Care Meeting.

Read full story

140+ Organisations Call on Government to put Children at the Heart of the Nation's Recovery

09 June 2021 / Posted in: Information, Support

Despite the Prime Minister’s pledges on Building Back Better and Levelling Up, the resignation of Sir Kevan Collins has highlighted a disturbing truth: supporting babies, children and young people to recover from the impact of the pandemic is still not a priority for Government investment. That’s why we’re joining the National Children’s Bureau and 140+ organisations in a call to Government to put babies, children and young people at the heart of COVID-19 recovery.

Read full story

How Scales and Measurements Will Work Now for England’s MAA for Nusinersen

04 June 2021 / Posted in: Treatments & Research

The nusinersen MAA requires data to be collected about each individual person being treated. This is so that you and your clinical team can monitor if the treatment is working for you / your child – bearing in mind that stabilisation of your / your child’s condition is a key aim. Here we explain how the scales and measurements will work going forwards.

Read full story

Evidence and Summary that led to NICE’s Type 3 Access to Nusinersen Decision Published

04 June 2021 / Posted in: Treatments & Research

Today you can read what sat behind this momentous decision first announced on 4th May. Over almost two years, SMA UK worked hard to advocate and contribute to this, amplifying the voices of the families and adults who have been impacted so distressingly by their exclusion from treatment.

Read full story

NICE publishes its Final Evaluation Document on Zolgensma

04 June 2021 / Posted in: Treatments & Research

Today, NICE confirmed its recommendations originally announced on 8th March 2021. There is now an appeal period closing on 18th June. Final publication is scheduled for 7th July 2021.

Read full story

Update from the National Multidisciplinary Team (NMDT) on the progress of the Zolgensma Treatment Programme in England

03 June 2021 / Posted in: Treatments & Research

This newly formed team of lead consultants from the four infusion centres is meeting very regularly. They are working on the many operational aspects of this new national programme that need to be in place to ensure that it is delivered in the best most equitable way possible.

Read full story