Our Research Community

FightSMA invests in a broad range of promising research efforts, critically reviewing the full spectrum of scientific possibilities until an effective treatment and cure is found. Every year in April, FightSMA brings together the world’s leading SMA scientists to discuss unpublished research and educate families about possibilities in the pipeline and where promising research is headed.
Chris Lorson

Chris Lorson, Ph.D.

FightSMA Scientific Director

Professor, Veterinary Pathobiology
University of Missouri

Adrian Krainer

Adrian Krainer, Ph.D.

Professor, Cold Spring Harbor Laboratory

Alex MacKenzie

Alex MacKenzie, MD, Ph.D.

FightSMA Scientific Advisory Co-Chair

Professor and Senior Scientist, Pediatrics,

Allison Ebert

Allison Ebert, Ph.D.

Assistant Professor,
Department of Cell Biology, Neurobiology & Anatomy,
Medical College of Wisconsin

Arthur Burghes

Arthur Burghes, Ph.D.

Professor, Molecular and Cellular Biochemistry, The Ohio State University

Brian Kaspar

Brian Kaspar, Ph.D.

Principle Investigator, Associate Professor,
Nationwide Children’s Hospital, The Ohio State University

Cathleen Lutz

Cathleen Lutz, Ph.D.

Repository Director and Research Scientist, Genetic Resources, The Jackson Laboratory

Charlotte Sumner

Charlotte Sumner, MD

Associate Professor,
Neurology and Neuroscience, Johns Hopkins School of Medicine

Christine DiDonato

Christine DiDonato, Ph.D.

Associate Professor,
Department of Pediatrics,
Ann & Robert H. Lurie Children's Hospital, Northwestern University

Clive Svendsen

Clive Svendsen, Ph.D.

Director, Regenerative Medicine Institute, Department of Biomedical Sciences, Cedars-Sinai Medical Center

Conrad Gilliam

Conrad Gilliam, Ph.D.

Dean for Research and Graduate Education, Biological Sciences Division, Department of Human Genetics, The University of Chicago

Douglas Kerr

Douglas Kerr, MD, Ph.D., MBA

Senior Director of Neurology Research and Development, Clinical Development, Biogen Idec

Elliot Androphy

Elliot Androphy, MD

Professor and Chair,
Department of Dermatology, Indiana University School of Medicine

Frank Bennett

Frank Bennett, Ph.D.

Senior Vice President of Research, Ionis Pharmaceuticals

Friedrich Metzger

Friedrich Metzger, Ph.D.

Head of Discovery Rare Diseases, Pharma Research & Early Development Neuroscience, F. Hoffmann-La Roche Ltd.

John Bach

John Bach, MD

Professor of Neurosciences,
Department of Physical Medicine and Rehabilitation, Rutgers New Jersey Medical School

John Porter

John Porter, Ph.D.

Program Director,
Extramural Research Program, National Institute of Neurological Disorders and Stroke-National Institutes of Health

Judith Melki

Judith Melki, MD, Ph.D.

Professor of Medical Genetics, University of Paris

Kathie Bishop

Kathie Bishop, Ph.D.

Vice President of Clinical Development, Ionis Pharmaceuticals

Kathryn Swoboda

Kathryn Swoboda, MD

Professor, Neurology,
University of Utah

Kenneth Fischbeck

Kenneth Fischbeck, MD

Distinguished Investigator,
National Institute of Neurological Disorders and Stroke, National Institutes of Health

Lee Rubin

Lee Rubin, Ph.D.

Professor/Director of Translational Medicine, Stem Cell and Regenerative Biology, Harvard University

Livio Pellizzoni

Livio Pellizzoni, Ph.D.

Assistant Professor,
Pathology and Cell Biology, Columbia University

Mimoun Azzouz

Mimoun Azzouz, Ph.D.

Director of Research & Innovation, Department of Neuroscience, University of Sheffield

Monique Lorson

Monique Lorson, Ph.D.

Associate Research Professor, Department of Veterinary Pathobiology, University of Missouri

Nanci Yuan

Nanci Yuan, MD

Clinical Associate Professor, Pediatrics - Pulmonary Medicine, Stanford School of Medicine

Nicholas Boulis

Nicholas Boulis, MD

Associate Professor,
Department of Neurosurgery, Emory University

Nikolai Naryshkin

Nikolai Naryshkin, Ph.D.

Director, Biology,
PTC Therapeutics

Rashmi Kothary

Rashmi Kothary, Ph.D.

Deputy Scientific Director and Senior Scientist,
 Regenerative Medicine Program, Ottawa Hospital Research Institute

Robert Leshner

Robert Leshner, MD

Professor Emeritus,
Department of Neurosciences, University of California San Diego School of Medicine

Robin Reed

Robin Reed, Ph.D.

Professor and Principle Investigator, Department of Cell Biology, Harvard Medical School

Susan Iannaccone

Susan Iannaccone, MD

Professor, Pediatrics, Neurology & Neurotherapeutics, University of Texas Southwestern Medical Center

Tom Crawford

Tom Crawford, MD

Professor of Neurology and Pediatrics and Co-Director, Muscular Dystrophy Association Clinic

Neurology, Johns Hopkins

Anne Reitz, Ph.D.

Indiana University School of Medicine

Barrington Burnett, Ph.D.

Uniformed Services University of the Health Sciences

Constantin d’Ydewalle, Ph.D.

Johns Hopkins School of Medicine

Constatin d'Ydewalle

Constantin d'Ydewalle, Ph.D.

Postdoctoral Fellow,
Neurology, Johns Hopkins School of Medicine

Eileen Workman, Ph.D.

Columbia University

Erkan Osman, Pd.D.

University of Missouri

Faraz Farooq

Faraz Farooq, Ph.D.

Children’s Hospital of Eastern Ontario Research Institute

Hans Rindt

Hansjorg Rindt Ph.D.

Research Assistant Professor, Veterinary Pathobiology and Molecular Microbiology & Immunology

University of Missouri

Hansjorg Rindt, Ph.D.

University of Missouri

Kevin Foust, Ph.D.

The Ohio State University

Lyndsay Murray

Lyndsay Murray, Ph.D.

Lecturer in Anatomy, Integrative Physiology, The University of Edinburgh

Lyndsay Murray, Ph.D.

University of Edinburgh

Lyndsay Murray, Ph.D.

University of Edinburgh

Melissa Bowerman, Ph.D.

University of Oxford

Sara Custer

Sara Custer, Ph.D.

Indiana University School of Medicine

Sara Gombash, Ph.D.

The Ohio State University

Sarah Tisdale, Ph.D.

Columbia University

Steve Crone

Steven Crone, Ph.D.

Assistant Professor, Neurosurgery and Developmental Biology,
Cincinnati Children’s Hospital Medical Center

Steven Crone, Ph.D.

Cincinnati Children’s Hospital