Latest News: Research
12 August 2016 / Posted in: Research
In this article, Dr. Alex Murphy describes the sensitivity of discussions around 'end of life care'. He also introduces some of the studies that have been carried out to examine the effectiveness of palliative care.
02 August 2016 / Posted in: Research
Biogen and Ionis Pharmaceuticals have announced that Nusinersen has met the primary endpoint in an interim analysis of the phase 3 ENDEAR Study in infants with Infantile-Onset SMA.
28 July 2016 / Posted in: Research
The US Food and Drug Administration (FDA) has granted “Breakthrough Therapy” status to AVXS-101, a gene therapy drug with future potential to treat SMA.
07 July 2016 / Posted in: Research
Dr Haiyan Zhou discusses how gene therapy using harmless viruses called AAVs has great potential as a therapeutic approach to replace defective genes that are responsible for conditions such as SMA and Duchenne muscular dystrophy.
30 June 2016 / Posted in: Research
The Scoliosis Research Priority Setting Partnership wants to hear the views of people of all ages with scoliosis, their family, friends and health professionals.
20 June 2016 / Posted in: Research
The study is looking for participants to help increase understanding of the lived experience of SMA. It aims to support pharmaceutical companies with their research relating to potential treatments in SMA and increase the knowledge of SMA amongst the wider scientific community.
02 June 2016 / Posted in: Research
Safety concerns have prompted the pharmaceutical company Novartis to pause enrolment in the Phase 2 clinical trial of their SMN2 splice-modifying drug LMI070.
26 May 2016 / Posted in: Research
Dr. Alex Murphy, our Clinical Care Research Correspondent, looks at medications that target other conditions that have been investigated as a treatment for Spinal Muscular Atrophy.
23 May 2016 / Posted in: Research
At two separate conferences in the USA in May, Avexis has presented the latest data from the ongoing Phase 1 clinical trial of their gene therapy drug for SMA, AVXS-101.
11 May 2016 / Posted in: Research
In February this year, clinical researchers and patient representatives from Europe and the US met to start work on the major review of the International Standards of Care for SMA.