Access to Zolgensma Survey and Submission Sent to NICE
08 August 2019 / Posted in: Treatments & Research
Thanks to all who responded to our survey asking for your views on access to Zolgensma for infants with SMA Type 1. We've passed them on to MDUK & TreatSMA and used them in our joint submission with MDUK. Click here to see the results and our submission to NICE.
Biogen’s Community Statement Answers Some Questions
07 August 2019 / Posted in: Treatments & Research
SMA UK, MDUK and TreatSMA jointly asked Biogen a number of questions relating to access to the nusinersen Managed Access Agreement with NHS England, and access in the UK in general.
Potential SMA Treatment Granted Orphan Medicinal Product Designation By The European Medicines Agency (EMA)
31 July 2019 / Posted in: Treatments & Research
Cytokinetics announced that EMA has granted Orphan Medicinal Product Designation to reldesemtiv for the potential treatment of SMA. Reldesemtiv is a drug that has been shown, in the laboratory, to increase the force generated by skeletal muscle and delay the onset and extent of muscle fatigue.
NICE Publishes Final Guidance Following Its Appraisal Of Nusinersen
24 July 2019 / Posted in: Treatments & Research
As there have been no appeals, access to the treatment will be as outlined in the Managed Access Agreement. We have not forgotten those who don't currently have access and will continue to work to change this.
Registration for SMA Europe’s 2nd International Scientific & Clinical Congress on SMA Now Open
19 July 2019 / Posted in: Treatments & Research
The conference will be held at Génocentre in Evry, south of Paris between 5th & 7th February 2020 and is open for healthcare professionals and researchers in SMA to attend. The congress will feature a series of talks and workshops covering key scientific and clinical research fields in SMA.
NCB Survey: Experiences Of Using SEND Information, Advice and Support Services
18 July 2019 / Posted in: Information, Support
The National Children’s Bureau are carrying out a survey about people’s experience of using SEND information, advice and support services. Your answers to the questions will help the Government understand how good the services are.
Cardiff Airport Invites Passengers To Join PRM & Disability Forum
11 July 2019 / Posted in: Information
On 4th September, Cardiff Airport is holding a meeting to explore ideas, and to agree the scope for their proposed Passengers with Reduced Mobility (PRM) and Disability Forum. Members of the public and third sector organisations are invited to attend.
Contact's Resources For A Stress-Free Summer
11 July 2019 / Posted in: Information, Support
The summer holidays are very nearly upon us and Contact for Families with Disabled Children have put together their top tips for making summer as stress-free and fun as possible – this includes a link to their guide “Holidays, Play and Leisure”.
Response from NICE to the SMA Community re: NICE's Communications About Access to Nusinersen
10 July 2019 / Posted in: Treatments & Research
We have received this reply in response to the letter sent by SMA UK, MDUK and TreatSMA on 14th June 2019.
NICE / NHS England announce changes to the nusinersen Managed Access Agreement
03 July 2019 / Posted in: Treatments & Research
The MAA for nusinersen (Spinraza) will now include paediatric patients who have recently (in the previous 12 months) lost the ability to walk independently. It also commits to considering any significant evidence being made available by Biogen in relation to all with SMA Type 3 who are non-ambulant, that may impact on the eligibility criteria of the MAA. These are just two changes following many confidential discussions which we have been unable to report on until now.
