Cure SMA Announces 2016 Family Friendly Poster Session Presenters

The Family Friendly Research Poster Session is one of the most anticipated events of our Annual SMA Conference. Each year, Cure SMA invites researchers from the SMA Researcher Meeting, which is held in parallel with the family conference, to present their findings to families in way that is accessible to them.

During the event, families are encouraged to rotate around to the different posters to ask questions and learn from the researchers about the specific projects being presented. Many of the presenters are Cure SMA grantees, meaning that families can interact directly with the researchers who are receiving the funds they have raised or donated.

This year’s event will be held on Friday, June 17 at 7:00PM in the Center Ballroom. It will feature 30 presenters representing different SMA research projects. All of the clinical stage drug programs for SMA will be included among the presenters, plus a variety of basic research projects and clinical care research projects.

Presenters and Posters

  • AveXis INC. on “An innovative approach to spinal muscular atrophy: Replacement of the SMN1 gene with gene therapy”.
  • F. Hoffmann-La Roche and Genentech on “Roche SMA program – our commitment to patients and families”.
  • F. Hoffmann-La Roche on “An oral SMN2 splicing modifier in clinical development for patients with SMA”.
  • Cytokinetics on “CK-2127107, a selective activator of the fast skeletal muscle troponin complex, for the potential treatment of SMA”.
  • California Institute on Biomedical Research (CALIBR) on “Identification of drug candidates for SMA using genome-edited neuronal cells”.
  • RaNA Therapeutics on “A novel epigenic approach to treat SMA.”
  • Ionis Pharmaceuticals on “Developing Nusinersen: an antisense drug for the potential treatment of SMA”.
  • Chien-Ping Ko PhD, Charlotte Sumner MD, and Sergey Paushkin PhD on “A new book on SMA: Spinal Muscular Atrophy: Disease Mechanisms and Therapy”.
  • The Novartis on “Initial findings from the First-in-Human trial of the Splice Modulator LMI070 in Type 1 Spinal Muscular Atrophy (SMA)”.
  • Burghes Lab at Ohio State University on “Neuromuscular impact of symptomatic SMN restoration and development of assays for potency of scAAV9-SMN as well as identification of suppressors of SMA mutations”.
  • The Beattie Lab at Ohio State University on “Going fishing! What fish motor neurons can tell us about SMA”.
  • The Sumner Lab at Johns Hopkins University on “Long Non-Coding RNAs: Expanding SMA therapeutic targets”.
  • The Cléry Lab from ETH Zurich on “Splicing regulation in SMA”.
  • The Kothary Lab from Ottawa Hospital Research Institute on “Differential impact of SMA severity on disease: muscle as an example”.
  • The Côté Lab from the University of Ottawa on “SMN and friends: What we can learn from SMN interactors”.
  • Melissa A. Alderfer PhD at Nemours/Alfred I. duPont Hospital for Children on “Systematically assessing the psychosocial needs of families of children with SMA”.
  • Tariq Rahman MD at Nemours/Alfred I. duPont Hospital for Children on “WREX- A dynamic arm orthosis for children with SMA”.
  • The Androphy Lab at University of Indiana on “Developing novel therapies – a one-two knockout punch to SMA”.
  • The Sahin Lab at Harvard University on “microRNA biogenesis in spinal muscular atrophy”.
  • The Lotti Lab at Columbia University on “SMN-dependent cellular pathways and their contribution to SMA etiology”.
  • Chad Heatwole MD at University of Rochester on “Adults Living With SMA: A Patient-Centered Assessment of the Symptoms That Matter Most”.
  • Linda Lowes PhD at The Research Institute At Nationwide Children’s Hospital on “Tracking movement with the Microsoft Kinect “.
  • The Murray Lab at the University of Edinburgh on “When Do Motor Neurons Get Sick in SMA”.
  • Nilesh Mehta MD at Boston Children’s Hospital on “Body composition and individualized nutrition in pediatric SMA”.
  • Rebecca Hurst Davis MS, RD, CSP, CD at the University of Utah on “Diet and blood sugar in children with SMA type II”.
  • Deborah Boroughs RN MSN at BAYADA Home Health Care Bayada on “Bridging a care delivery gap for family caregivers of children with SMA types I and type II”.
  • Holly Peay PhD from Parent Project Muscular Dystrophy and RTI International on “To participate or not to participate? Decision making for SMA clinical trials”.
  • Stephen J. Kolb MD PhD at Ohio State University on “SMA infant biomarker study – super baby progress in 2016”.
  • The DiDonato Lab at Lurie Children’s Hospital of Chicago on “Modifying SMA phenotypes in milder SMA mice”.
  • Manuel Prieto Chief Technology and Innovation Officer at Marsi Bionics on “A wearable gait exoskeleton for the daily life activity of children with SMA”.

About the Annual SMA Conference

Every year, Cure SMA sponsors a conference to bring together the leading SMA researchers, clinicians, and families living with SMA. Cure SMA has been hosting the Annual SMA Conference since 1988. We look forward to reuniting as a community at this year’s conference in order to lend each other support and strength and to learn about the latest advances in research and care.

As always, the Family and the Researcher Conferences run alongside each other. Through the conferences, we bring together researchers, healthcare professionals, and families to network, learn, and collaborate. The Annual SMA Conference is the largest conference in the world for those affected by SMA, and also for those involved in providing support and care for SMA patients.

Our thanks to Biogen for their generosity as the presenting sponsor of the 2016 Annual SMA Conference.

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