Open Style Lab: Research Study About How Design Impacts Disability
19 July 2021 / Posted in: Information
Open Style Lab (OSL) is an award-winning nonprofit organisation in the US with a mission to make style and fashion accessible. They are currently running a longitudinal research study about how design impacts disability and are looking for female participants to be interviewed.
Two Job Opportunities at Pathfinders Neuromuscular Alliance
16 July 2021 / Posted in: Information, Support
One is Project Lead (developing Personal Assistance and Neuromuscular Conditions: A training programme for PAs). The other is Executive Assistant to CEO. Application deadline for both is Friday 23rd July.
DCP Campaign - Left Behind Report & Public Letter to the Chancellor
16 July 2021 / Posted in: Information, Support
The Disabled Children's Partnership has launched a new report - Left Behind - featuring freedom of information research as well as the results of their last survey of parents / carers. In conjunction, they have also launched a new campaigning action – a parent-led open letter to the Chancellor. Read more and sign the letter.
NHS England and NHS Improvement: UK Framework Action Plan Discussion
09 July 2021 / Posted in: Information, Treatments & Research
Since the publishing of The UK Rare Disease Framework on 9th January 2021, NHS England and NHS Improvement have been busy developing an Action Plan. Now, they and Findacure invite you to share your thoughts in a two-hour session on Tuesday 20th July.
Apitegromab Receives Fast Track Designation in the US
07 July 2021 / Posted in: Treatments & Research
Apitegromab (also known as SRK-015) is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by people living with SMA. Fast Track status is granted to drugs that show considerable potential for being able to treat life-threatening conditions that have limited therapeutic options.
NICE’s Guidance for NHS-Funded Access to Zolgensma Published
07 July 2021 / Posted in: Treatments & Research
Today, NICE’s recommendations for NHS-funded access to Zolgensma that were first announced in March, have now been published and finalised. These will continue to run in parallel to the additional agreement also made in March by NHS England. Thanks to the many stakeholders who have got us to this positive place.
Sign the Petition to Introduce Newborn Screening for SMA in the UK
25 June 2021 / Posted in: Information, Treatments & Research
SMA UK is a member of the UK SMA Newborn Screening Alliance which brings together patient groups, clinicians and many other stakeholders determined to see newborn screening for SMA introduced as part of the heel prick blood test in the UK. Show your support for our work and the work of the UK SMA Newborn Screening Alliance by signing the petition.
UK SMA Patient Registry Update - June 2021
25 June 2021 / Posted in: Treatments & Research
The UK SMA Patient Registry collects clinical and genetic information from individuals with Spinal Muscular Atrophy. Patient Registry Project Manager, Lindsay Murphy, has put together an update on the latest figures and updates about the registry.
SMA UK and Many Others Respond to NICE’s Consultation on Access to Risdiplam
23 June 2021 / Posted in: Treatments & Research
Our thanks to the many people we talked with who planned to make their own personal responses. These will powerfully echo the voices of those who responded to our surveys and spoke up at the committee meeting. Together, we are calling for access for all for whom risdiplam is a clinically safe option.
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.
