Researcher Mtg Event Title

22nd International SMA Researcher Meeting

June 14 - 16, 2018
Hilton Anatole Hotel | Dallas, TX

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Agenda

Special Session for 22nd Annual SMA Researcher Meeting

Clinical and Basic Questions of SMA in the Era of SMN Repletion: Challenge and Opportunity 

The advent of an FDA approved SMN enhancing drug has ushered in a new stage in the SMA community. As we celebrate the long awaited first drug approval, we know there remains much to be done. While Spinraza offers tremendous benefit to those with SMA, there is also a need for continued research. The development of additional therapies to be used independently or in combination with SMN enhancing approaches requires understanding more about SMA biology and disease mechanisms. Studying the "new" SMA phenotype after treatment will unveil new, unmet patient need, perhaps including extra-neuronal phenotypes and the need for such therapies. The goal of this session is to discuss these new challenges and opportunities. The session begins by exploring the efficacy and unmet needs of current treatment and is followed by investigating patient phenotypes post treatment. Next, the neurodevelopmental need for prenatal SMN is discussed and its requirement for complete restoration of phenotype. Following that will be a discussion of the unanswered basic biology questions pertaining to SMA and their implications for therapy development. The panel discussion will focus on prioritizing future research endeavors to address unmet medical need for SMA patients. Moderator, Inviting Richard Finkel, MD, Chief, Division of Neurology, Nemours Children's Hospital

8:35 AM Exploring Unmet Medical Need in SMA: Comparing SMN Enhancing Efficacy in Pre-symptomatically Versus Symptomatically Treated Patients Thomas Crawford MD, Professor of Neurology and Pediatrics, Johns Hopkins School of Medicine

9:00 AM Unmet Medical Need in Adults and Teens with SMA John Day, MD, PhD, Professor, Neurology & Neurological Sciences Director, Stanford Neuromuscular Disorders Program, Stanford University Medical Center

9:25 AM Overview of Therapies in Development: What are the Current Gaps in the SMA Drug Development Pipeline? Stephen Kolb, MD, PhD, Director, ALS/MND Multidisciplinary Clinic and Translational Research Program, Associate Professor, Department of Neurology and Department of Molecular & Cellular Biochemistry, Ohio State University

9:50 AM Extra-neural and Peripheral Phenotypes of SMA Speaker to be determined

Break

10:50 AM Is there a Developmental Component to SMA: Is SMN Required Prenatally? Charlotte Sumner, MD, Professor of Neurology, Johns Hopkins School of Medicine

11:15 AM SMA Biology: What we know and what we need to know in our understanding of SMA? Arthur Burghes, PhD, Professor of Molecular and Cellular Biochemistry, Ohio State University

11:35 AM The role of SMN at the NMJ: current knowledge and remaining questions. Umrao Monani, PhD, Associate Professor of Pathology and Cell Biology, Columbia University

12:00 PM Panel Discussion

12:30 PM Lunch

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