Latest News: Research
17 January 2017 / Posted in: Research
The US Food and Drug Administration (FDA) has granted Orphan Drug Status to RG7916, a splice-modifying drug developed through a partnership between Roche, PTC Therapeutics, and the SMA Foundation.
12 January 2017 / Posted in: Research
The first step towards possible licensing of Spinraza in the UK is for it to go through the EMA appraisal process. Read the joint letter sent by UK SMA charities asking the EMA to speed this up. Download a template letter if you want to write to your own MP.
11 January 2017 / Posted in: Research
A potential new therapy for SMA, which could possibly be combined with other drugs such as Spinraza to enhance clinical effectiveness, has been identified and successfully tested in mouse models of the disease.
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.