NICE Asks for Comments on its Appraisal Consultation Document (ACD) for Risdiplam
02 June 2021 / Posted in: Treatments & Research
NICE’s initial position is to say 'no' to approving risdiplam for NHS funding. This is disappointing news, but not unusual at this stage, and NICE is asking for comments on this by 23rd June, which they will review at their next committee meeting on 13th July. We will be responding to the consultation paper, again advocating for access for all as soon as possible. We remain hopeful that this process is similar to other appraisals whereby once more work is completed, the initial 'no' changes to ‘yes’.
First Baby in England Treated with Zolgensma
01 June 2021 / Posted in: Treatments & Research
It’s very heartening to see the first child with SMA Type 1 being treated with this ground-breaking gene therapy via the NHS at Evelina Hospital. As we support families through this understandably anxious period, we are doing all we can to ensure that those currently waiting will, as soon as possible, be able to have full discussions with their clinical team to see if this treatment option will also be appropriate for their child.
Invitation to Parliamentary Meeting on Newborn Screening
28 May 2021 / Posted in: Information, Treatments & Research
On 9th June, the APPG on Rare, Genetic and Undiagnosed Conditions will be holding a meeting on newborn screening. The Newborn Screening Alliance is working with Genetic Alliance UK to improve access to newborn screening. You can ask your MP to attend: appg@geneticalliance.org.uk
Access to Zolgensma – Latest Updates on the Q&As for England
26 May 2021 / Posted in: Treatments & Research
We have received some further answers from NHS England for our Q&As. We will continue to update these as soon as we receive new information, giving answers to the many questions people have asked.
Protecting Disabled People From Fire
25 May 2021 / Posted in: Information
Following a request from a member of the SMA Community, we added our support to this open letter from Disability Rights UK to the Government requesting urgent action to: protect the lives of disabled people in the event of fire; prevent disabled leaseholders being forced to leave adapted homes; ensure that disabled leaseholders are not impacted by the costs of remedial safety works.
Important Risdiplam Access Updates
21 May 2021 / Posted in: Treatments & Research
Risdiplam now has GB marketing authorisation, but there’s still a long way to go before we know NICE's recommendation on NHS funding. Also, good news that Roche will submit an application in June for NHS provision in Scotland. The EAMS scheme for people already enrolled continues, but closes to new people, unless they've been identified before the announcement. Anyone not yet enrolled who thinks they should be eligible should now contact their clinical team.
Online Event: Periods and Disability, Impairments & Conditions
20 May 2021 / Posted in: Information, Support
Organised by environmental non-profit organisation City to Sea, this online event is taking place on Friday 28th May (Menstrual Health Day). The panel will be discussing periods, menstrual education and period products, through the lens of disability, impairments and conditions.
Living With SMA Podcast - Episode #4 Out Now!
20 May 2021 / Posted in: Information, Support
In this episode - 'Adult Onset SMA' - SMA UK Host Martyn Sibley chats with Peter, who shares his personal journey to a diagnosis of SMA Type 4 in adulthood. Talking about how this ‘Adult Onset’ form of the condition has changed his life, Peter describes some of the challenges he’s faced and overcome and how he’s adapted to living with SMA.
Zolgensma Infusion Centres Announced
14 May 2021 / Posted in: Treatments & Research
We’re pleased that NHS England and NHS Improvement have shared this today to keep the Community up to date with developments. Click through to see the update and list of centres.
Which Centres are Providing Nusinersen and / or Risdiplam?
12 May 2021 / Posted in: Treatments & Research
Following further enquiries, we have summarised the information from NHS England’s 10th May notice about this in a table that may be easier to read. You will need to check what your Centre’s position is in terms of the guidelines that have been issued.
