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Latest News: Research

Roche to buy Trophos

23 January 2015 / Posted in: Research

Roche, the Swiss pharmaceutical giant, has agreed to purchase the small, late-stage clinical drug testing company, Trophos, and its lead drug with potential for SMA, Olesoxime

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SMN Gene Therapy in a Large SMA Model

06 January 2015 / Posted in: Research

Prof. Arthur Burghes (Ohio State University) and colleagues have just published a study aimed at determining whether reducing SMN levels in pig motor neurons results in symptoms similar to SMA.

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Isis Pharmaceuticals Initiates Second Phase III Trial of ISIS-SMNRX

12 December 2014 / Posted in: Research

ISIS Pharmaceuticals has announced the start of a second Phase III trial of its gene therapy drug ISIS-SMNRx.

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PTC Therapeutics SMA drug enters Phase Ib/IIa trial

26 November 2014 / Posted in: Research

PTC Therapeutics has announced that its SMN2 splice-modifying drug, RG7800, has entered a Phase Ib/IIa clinical trial in adults and children with SMA.

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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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