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Latest News: Research

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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ReGenX Reach Deal With AveXis To Advance Potential SMA Gene Therapies

15 April 2014 / Posted in: Research

ReGenX Biosciences, LLC and ...

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New Therapeutic Target For SMA Identified

20 March 2014 / Posted in: Research

Research led by ...

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Trophos Announces Positive Olesoxime Phase II Trial Results

11 March 2014 / Posted in: Research

The French pharmaceutical company Trophos has ann...

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Isis Pharmaceuticals Announces Interim Results Of ISIS-SMNRx Phase II Trials

03 March 2014 / Posted in: Research

ISIS Pharmaceuticals has provided an update...

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Friday 28th February is Rare Disease Day

24 February 2014 / Posted in: Research

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