Latest News: Research
06 January 2017 / Posted in: Research
In this timely article, Dr. Alex Murphy describes the organisations involved and outlines the processes that have to be gone through before a drug can be prescribed for an individual in the UK.
04 January 2017 / Posted in: Research
Read a joint statement from SMA Support UK, Muscular Dystrophy UK and The SMA Trust.
22 December 2016 / Posted in: Research
Read a statement from Roche about their 2017 clinical trials of drug RG7916.
21 December 2016 / Posted in: Research
Read the report on progress towards an Expanded Access Programme of the ENDEAR trial of nusinersen which treats eligible infants with Spinal Muscular Atrophy Type 1.
15 December 2016 / Posted in: Research
Results from a phase 2 clinical trial of the Ionis Pharmaceuticals gene therapy drug, Nusinersen (now marketed as Spinraza), have been published in the medical journal The Lancet.
06 December 2016 / Posted in: Research
In Dr. Alex Murphy's latest article, he summaries some of the more common CATs (Complementary and Alternative Therapies) and looks into evidence for their use in SMA or similar conditions.
05 December 2016 / Posted in: Research
Applications are open for PhD Studentships and one of the available projects is 'Psychosocial and ethical issues in perinatal gene and cell therapies for Spinal Muscular Atrophy'. Deadline for applications if Friday 9th December.
08 November 2016 / Posted in: Research
Biogen and Ionis Pharmaceuticals have provided the following community statement regarding the interim analysis of the CHERISH trial.
07 November 2016 / Posted in: Research
AveXis has announced details about a future pivotal trial of their gene therapy drug for SMA Type 1, AVXS-101.
31 October 2016 / Posted in: Research
Biogen have announced that their New Drug Application (NDA) for Nusinersen has been accepted by the US Food and Drug Administration (FDA) for Priority Review.