Latest News: Research
01 June 2017 / Posted in: Research
Families with eligible children with SMA Type 1 currently under Great Ormond St Hospital’s care, who wish to access the nusinersen EAP, will be able to do so over the next 3 months. This includes children under GOSH’s care who currently access treatment in Europe.
30 May 2017 / Posted in: Research
We are working with Muscular Dystrophy UK, the SMA Trust and TreatSMA. Read about our focus now and how you can help.
25 May 2017 / Posted in: Research
Cytokinetics has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to its compound CK-2127107 for the potential treatment of SMA.
16 May 2017 / Posted in: Research
Biogen responds to NHS England's requests about the UK EAP for infants with SMA Type 1; they also tell us when they hope to hear from NICE.
11 May 2017 / Posted in: Research
We asked about the position in the UK and overseas.
10 May 2017 / Posted in: Research
You can now read the latest information about NHS England’s position on considering the possibility of supporting the costs of delivering the EAP in England. We now have authority to share information from the meeting we attended on 25th April with the NHS England Specialised Commissioning team.
26 April 2017 / Posted in: Research
Cytokinetics has announced that it has initiated enrolment of a second cohort of SMA patients with SMA Types 2, 3 or 4 in their phase 2 clinical trial of CK-2127107, in North America.
25 April 2017 / Posted in: Research
Biogen and Ionis Pharmaceutical have released information on the CHERISH and NURTURE clinical trials of their gene therapy drug, Spinraza (formerly nusinersen).
21 April 2017 / Posted in: Research
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the marketing approval of nusinersen.
18 April 2017 / Posted in: Research
This new research article looks at recent research into different aspects of spinal surgery, in the treatment of scoliosis in Spinal Muscular Atrophy.