Cure SMA Awards $140,000 Grant to Oliver Gruss, PhD, Rheinische Friedrich-Wilhelms-Universitat Bonn and Utz Fischer, PhD, Julius-Maximilians-Universitat Wurzburg, Germany

Cure SMA has awarded a $140,000 research grant to Drs. Oliver Gruss and Utz Fischer at Rheinische Friedrich-Wilhelms-Universitat Bonn and Julius-Maximilians-Universitat Wurzburg in Germany to study “Regulatory cues modulating the activity of SMN in human cells.” This projects aims to understand the details of how SMN protein works in the cell. Drs. Gruss and Fischer […]

Cure SMA Awards $140,000 Grant to Oliver Gruss, PhD, Rheinische Friedrich-Wilhelms-Universitat Bonn and Utz Fischer, PhD, Julius-Maximilians-Universitat Wurzburg, Germany Read More »

Cytokinetics Receives FDA Orphan Drug Designation for CK-2127107 for SMA

Cytokinetics, Inc. recently announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CK-2127107, a next-generation fast skeletal muscle activator, for the potential treatment of spinal muscular atrophy. Orphan designation is granted to drugs and biologic products that are intended for the safe

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Cure SMA Announces 2017 Family Friendly Poster Session Presenters

We’ve recently seen significant advances in SMA research come to fruition, and with 17 drug programs in development, including five in clinical trials, we are invested to treating all ages, stages and types of SMA. With such great promise in the research landscape, we know that collaboration between academics, industry, government and families is crucial.

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Cure SMA Awards $140,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires, Argentina

Cure SMA and FAME (Families of SMA, Argentina) have awarded a $140,000 research grant to Alberto Kornblihtt, PhD, at the Universidad de Buenos Aires, Argentina, for his project, “Epigenetics in SMN2 E7 Alternative Splicing.” Epigenetics refers to changes that affect how much protein is produced from each gene, without altering the DNA sequence. Dr. Kornblihtt

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RUSP Nomination for SMA Accepted into Evidence Review

The Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (SACHDNC) announced that they have accepted spinal muscular atrophy into the review process for the Recommended Uniform Screening Panel (RUSP). The RUSP is a list of conditions that all newborns in the US are recommended to be screened for. The RUSP application for SMA

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Cure SMA Welcomes Back Biogen as a National Premier Partner for 2017

Cure SMA is thrilled to welcome Biogen back as a National Premier Partner for 2017. The National Premier Partnership represents the highest level of support for the SMA community. As part of their partnership, Biogen will be sponsoring all walk-n-rolls, golf events, galas and awareness events across the US for the entirety of 2017. Biogen

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Cure SMA Awards $75,000 Grant to Yong-Chao Ma, PhD, Ann & Robert H. Lurie Children’s Hospital of Chicago

Cure SMA has awarded a $75,000 research grant to Yong-Chao Ma, PhD, Ann & Robert H. Lurie Children’s Hospital of Chicago, for his project, “Regulation of Motor Neuron Defects by Cdk5 Signaling in SMA”. Dr. Ma and his team have found that the activity of a protein called Cdk5, cyclin-dependent kinase 5, is significantly increased

Cure SMA Awards $75,000 Grant to Yong-Chao Ma, PhD, Ann & Robert H. Lurie Children’s Hospital of Chicago Read More »

Cure SMA Awards $75,000 Grant to Stephen J. Kolb, PhD, Ohio State University

Cure SMA has awarded a $75,000 research grant Stephen J. Kolb at Ohio State University, for his project, “Arrested Development or Neurodegeneration? An approach to understand developmental motor neuron pathology in SMA”. Dr. Kolb and his team will create a large animal model of newborn infants with SMA. They will use this model to understand

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Cure SMA Awards $75,000 Grant to Chris Lorson, PhD, University of Missouri

Cure SMA has awarded a $75,000 research grant to Chris Lorson, PhD, at the University of Missouri, for his project, “Examining the role of astrocytes and the influence upon lower motor neuron susceptibility in SMA.” SMA is caused by a mutations in the SMN1 gene. This gene is expressed in every cell in the body;

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Cure SMA Announces Increase in Basic Research Funding, Now Exceeding $1 Million in Grants

We are excited to announce that two additional grants have been added to this round of basic research funding, which brings the total to $1.03 million in grants for this year. We sincerely thank our incredible community and its growing support, which made this extra investment possible. Whether families raising funds and awareness, researchers investigating

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