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Latest News: Research

Eurordis (European Organisation for Rare Diseases) Needs Your Expertise

16 February 2016 / Posted in: Research

Eurordis is conducting a survey in an effort to better understand how well people are engaged in decisions about their medical care.

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Biogen and Ionis Pharmaceuticals Provide Update on Nusinersen (Formerly Known as ISIS-SMNRx)

31 January 2016 / Posted in: Research

Biogen and Ionis Pharmaceuticals (previously Isis Pharmaceuticals) have provided an update on the clinical development of their antisense oligonucleotide drug Nusinersen, which used to be known as Isis-SMNRx.

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Two New Clinical Care Research Articles

12 January 2016 / Posted in: Research

Dr. Alex Murphy, our Clinical Care Research Correspondent, has written two review articles; one about diet and SMA and one about exercise and SMA.

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Cytokinetics Initiates Phase II Trial of Potential SMA Drug

11 January 2016 / Posted in: Research

Cytokinetics and Astellas have begun enrolling SMA patients in a Phase II clinical trial of their muscle-activating drug, CK-2127107.

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Roche Provides Updates on New Compound and Moonfish Trial

11 January 2016 / Posted in: Research

Roche, PTC Therapeutics, and the SMA Foundation have announced that they are to begin testing a second splice-modifying drug with potential to treat SMA.

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Avexis Completes Phase I Trial Patient Enrolment

08 January 2016 / Posted in: Research

Avexis has announced that patient enrolment is complete in a Phase I clinical trial of their gene therapy drug, scAAV9.CB.SMN.

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Isis Pharmaceuticals changes its name to Ionis Pharmaceuticals

06 January 2016 / Posted in: Research

After almost a year of deliberation, Isis Pharmaceuticals has rebranded itself as Ionis Pharmaceuticals in order to avoid confusion and negativity associated with Islamic State, also commonly known as Isis.

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Blood Vessel Defects Contribute to SMA Motor Neuron Loss

17 December 2015 / Posted in: Research

New research from the laboratory of Simon Parson (University of Aberdeen) and collaborators in London and Oxford indicates that a faulty blood supply may play an important role in the death of motor neurons seen in spinal muscular atrophy.

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A New Scientist Q & A

16 November 2015 / Posted in: Research

Read the latest scientist Q & A. Being interviewed this time is Ewout Groen. Ewout was recently awarded a prestigious Wellcome Trust Fellowship for early career researchers.

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Isis Pharmaceuticals Initiates New ISIS-SMNRx Extension Study, SHINE

23 October 2015 / Posted in: Research

ISIS Pharmaceuticals has initiated a new open-label, extension study of their antisense oligonucleotide gene therapy drug ISIS-SMNRx.

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