Research
PTC Therapeutics Announces New Phase 1a/2b Clinical Trial
PTC Therapeutics, Inc. announced today that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) has started a […]
Read More ›Isis Announces Second Phase III Clinical Trial
Isis recently announced the start of CHERISH, a multi-center Phase III clinical study evaluating the efficacy and safety of the investigational compound, ISIS-SMNRx, in non-ambulatory […]
Read More ›Cure SMA Scientific Advisory Board Meets in Washington D.C.
Today and tomorrow, Cure SMA’s Scientific Advisory Board (SAB) will be meeting in Washington D.C. Our Scientific Advisory Board is a recognized group of experts […]
Read More ›Promising Results Published on Cure SMA-Funded Gene Therapy Project
A manuscript from the laboratory of Dr. Brian Kaspar of Nationwide Children’s Hospital was recently published in the journal Molecular Therapy. The paper, “Improving single […]
Read More ›Read the Fall 2014 Issue of Compass
The latest issue of Compass, our research publication, is now available online. This Fall 2014 issue includes news from our 2014 SMA researcher meeting, and […]
Read More ›2014 SMA Researcher Meeting Summary: Clinical Trials
We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. […]
Read More ›2014 SMA Researcher Meeting Summary: Therapy Development
We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. […]
Read More ›2014 SMA Researcher Meeting Summary: SMA Pathology
We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. […]
Read More ›2014 SMA Researcher Meeting Summary: The SMN Protein
We will be posting a series of summaries from our 2014 researcher meeting, highlighting some of the most interesting new developments and discoveries presented there. […]
Read More ›Isis Releases Phase II Clinical Trial Results
Today, Isis released results from their Phase II open-label clinical trials of ISIS-SMNRx. These results covered two separate trials: a trial in infants with SMA […]
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