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.

The Annual SMA Conference is one of the primary ways we encourage this collaboration, and the Family Friendly Poster Session is one of the most anticipated events within the conference.

Each year, Cure SMA invites researchers from the SMA Researcher Meeting to present their findings to families in an accessible way. This allows the researchers to see the impact of their work on families, and also gives families the opportunity to ask important questions, hear about the latest advances in research and meet the researchers they have fundraised for.

This year’s event will be held on Friday, June 30 at 7:00PM in the Fantasia Ballroom. It will feature 30 presenters, many of whose projects are Cure SMA funded,  and represent different SMA research projects. 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

Utz Fischer, PhD and Oliver Gruss, PhD from the University of Wuerzburg, Germany and the University of Bonn, Germany on “Regulation of SMN by post-translational modifications”.

Christine Beattie, PhD and Hao Le, PhD from Ohio State University on “The effect of low SMN on motoneuron development”.

Antoine Clery, PhD and Frederic H.T. Allain, PhD form ETH Zurich on “Splicing regulation in SMA”.

Marie-Therese Khairallah, PhD and Chris Lorson PhD from University of Missouri on “Dr. StrangeCell or: How I learned to stop worrying and love the astrocyte”

Marc-Olivier Deguise and Rashmi Kothary PhD from Ottawa Hospital Research Institute on “Is the immune system affected in SMA patients?”

Stacy Rudnicki, MD from Cytokinetics on “Clinical Trial Update of CY 5021: CK-2127107, an activator of skeletal muscle, for the potential treatment of Spinal Muscular Atrophy”.

Vanessa Christie-Brown from SMA Europe on “Disease impact on general well-being and therapeutic expectations of European Type II and Type III spinal muscular atrophy patients”.

Richard Finkel, MD and Kelly Wydronkowski from Nemours Children’s Hospital on “Implementation of SOC guidelines at Nemours”.

Tariq Rahman, PhD from Nemours/A.I. DuPont Hospital for Children on “WREX exoskeleton for children with SMA”.

Andreanne Didillon, Fatima Mostefai, Amir Haghandish from University of Ottawa on “SMN and friends: What we can learn from SMN interactors?”

Hikari Tanaka, Nikki McCormack, Mahlet Abera PhD, and Barrington Burnett PhD from the Uniformed Services University of the Health Sciences and The F. Edward Hébert School of Medicine on “Uncovering ways to slow SMN destruction”.

Lisa Belter, Jill Jarecki PhD, Kenneth Hobby, Cynthia Jones PhD, Suzanne Cook PhD, and Sandra Reyna MD from Cure SMA on “Cure SMA membership: Findings from the 2017 membership survey”.

Jackie Glascock PhD, Jill Jarecki PhD, Megan Lenz, and Kenneth Hobby on “Cure SMA newborn screening for spinal muscular atrophy: Federal and state implementation”.

John Jae Hong Park from Johns Hopkins University School of Medicine on “SMN expression is developmentally regulated in human spinal cord and muscle”.

Remy Bordonne, PhD from IGMM-CNRS on “Identification of the protective mechanism of a SMN modifier gene using S. pombe as model organism”.

Geoffrey Laff, PhD from Spotlight Innovation Inc. on “STL-182, an orally-available small molecule that stabilizes SMN protein”.

AveXis, Inc. on “AveXis: Committed to the development of therapies for patients affected by rare and life-threatening neurological genetic diseases”.

Sangeeta Jethwa MD, Karl Yen , Ksenija Gorni MD PhD, Anne Marquet PhD, Tim Seabrook PhD, Jeppe Buchbjerg, Bertrand Verwee, Michael Ostland PhD, Parul Houston MD, Paulo Fontoura MD, John Baird PhD, Karen Chen PhD, Mary-Frances Harmon, Nikolai Naryshkin PhD, Sergey Paushkin PhD, Omar Khwaja MD from F.Hoffmann-La Roche Ltd on “Roche SMA drug program for SMA”.

Rosangel Cruz, Kenneth Hobby, Jill Jarecki, PhD., Megan Lenz, and Lisa Belter from Cure SMA on “Externally-led patient focused drug development meeting for spinal muscular atrophy”.

Arnab Chatterjee, PhD from Calibr on “Innovative screening approaches to ID the next generation of SMN-inducing compounds”.

Arthur Burghes, PhD from The Ohio State University on “Sequence changes effecting severity of SMA and development of combined treatments”.

T Seabrook PhD, K Gelblin, K Gorni MD PhD, T Wiese MD, A Marquet PhD, C Czech PhD, D Kraus, D Trundell PhD, B Verwee, I Schwersenz MD, K Rucinski, N Gusset, S Braun PhD, J Baird PhD, K Chen PhD, M Harmon, N Naryshkin PhD, S Paushkin PhD, S Jethwa MD, from F.Hoffmann-La Roche Ltd on “Patient partnership in SMA”.

Jean Giacomotto, PhD from The Queensland Brain Institute on “Innovative genetic approach to recapitulate and study SMA in the zebrafish”.

Lawrence Charnas, MD, PhD and Emilie Voltz, PhD from Novartis on “Safety and efficacy findings in the first-in-human trial of the oral splice modulator branaplam in Type 1 spinal muscular atrophy (SMA) after two years”.

Yongchao Ma PhD from Northwestern University / Lurie Children’s Hospital of Chicago on “Targeting mitochondria, the powerhouse in motor neurons, for treating SMA”.

Chad Heatwole, MD from University of Rochester on “Gathering your opinion on treatment effectiveness: The creation and use of the SMA-Health Index”.

Kimberly Long, PhD, Micah Webster, PhD, Adriana Donovan PhD, Karen S. Chen PhD, Kelly Howell PhD, Sergey PaushkinPhD Ramzi Khairallah PhD, Karen O’Shea PhD, Nagesh Mahanthappa PhD, and Alan Buckler PhD from Scholar Rock on “Scholar Rock’s novel approach to strengthening muscle in SMA patients”.

Christine DiDonato, PhD, Kristin Kroschell, PhD, Nancy Kuntz MD, and Vamshi Rao MD from Anne & Robert H. Lurie Children’s Hospital/ Northwestern University. “The Lurie Children’s Hospital experience: Spiranza dosing across the age spectrum, quantitative assessment of function and efforts towards molecular markers and new therapies”.

Jacqueline Montes, PT, EdD, NCS and Sally Dunaway Young, PT, DPT from Pediatric Neuromuscular Clinical Research Network (PNCRN) on “Clinical Observations of Fatigue & Endurance in Spinal Muscular Atrophy”.

Stephen J Kolb, MD, PhD from The Ohio State University, Wexner Medical Center on “NeuroNEXT SMA Biomarker Study. Will present the natural history study and emphasize the importance of these types of studies when placebo arms in trails are not ethical”

Our thanks to Biogen for their generosity as the presenting sponsor of the 2017 Annual SMA Conference