Latest News: Treatments & Research
All Party Parliamentary Group on Access to Medicines and Medical Devices makes recommendations for the NICE Methods Review
25 September 2019 / Posted in: Treatments & Research
Representing the views of a broadly-based group of stakeholders (including SMAUK) this report will be taken to the Government, the NHS, and NICE, to continue dialogue about what should change to support better patient access to innovative therapies.
20 September 2019 / Posted in: Treatments & Research
Announced publicly at the 13th Congress of the European Paediatric Neurology Society (EPNS), the DEVOTE study will evaluate if higher dosing of nusinersen can provide improved efficacy in the treatment of SMA across a broad population of patients.
Physiotherapy - Based Measures (HINE, CHOP INTEND, HMFSE) for People with SMA and how they are used to Monitor Treatment Outcomes
02 September 2019 / Posted in: Treatments & Research
In this new article, our Clinical Care Research Correspondent, Dr Alex Murphy, summarises: the way some of the more commonly used physiotherapy-based outcome measures in SMA work; the research that shows how these measures would be expected to progress for those with SMA who have not accessed drug treatments.
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.
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.
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.
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.
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.
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.