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Latest News: Research

ISIS-SMNRx Phase II Trial Results Released

14 October 2014 / Posted in: Research

ISIS Pharmaceuticals has released detailed results from their Phase II clinical trials of the gene therapy drug ISIS-SMNRx.

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Isis Pharmaceuticals Begin Recruitment for Phase III Trial of ISIS-SMNRX

10 October 2014 / Posted in: Research

ISIS Pharmaceuticals have begun recruiting young SMA patients at a number of sites in the US for a Phase III trial of its gene therapy drug ISIS-SMNRx.

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SMA Support UK at the Cure SMA Conference 2014

10 October 2014 / Posted in: Research

The annual Cure SMA Conference (previously known as the Families of SMA Conference) was held at The Gaylord National Resort & Convention Center, Oxon Hill, Maryland‎, USA.

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Avexis Gene Therapy Gains Orphan Drug Status

08 October 2014 / Posted in: Research

ChariSMATM, a potential gene therapy program for SMA, has received Orphan Drug Designation from The US Food and Drug Administration (FDA).

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MRF Motor Neurone Research Competition

26 August 2014 / Posted in: Research

The Medical Research Foundation (MRF) is inviting applications for research that will increase understanding of the diseases and conditions affecting motor neurones.

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SMA Europe - Call for SMA Research Project 2014

26 August 2014 / Posted in: Research

SMA Europe is pleased to announce its 7th International Call for SMA Research Projects.

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New highly selective SMN2 modifying drugs identified

13 August 2014 / Posted in: Research

Researchers in the US have identified three new, orally available compounds that efficiently...

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Isis Pharmaceuticals Initiate Phase III Trial of ISIS-SMNRX

08 August 2014 / Posted in: Research

ISIS Pharmaceuticals has announced the begi...

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Genzyme Publishes SMN Gene Therapy Study

30 May 2014 / Posted in: Research

The US biotech company, Genzyme, has published new results in animal models supporting the use of harmless, genetically engineered viruses to treat SMA.

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SMN Gene Therapy Trial Initiated In The US

30 April 2014 / Posted in: Research

A phase I clinical trial of a potential gene therapy for SMA has opened for infants wit...

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