Leading SMA Researchers Attend 2016 FightSMA Annual Conference


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Phase 1 trial of SMA gene therapy

FightSMA, an all-volunteer, parent-led nonprofit dedicated to finding treatments for spinal muscular atrophy (SMA), celebrated its 25th anniversary with a research conference that attracted leading SMA researchers from all over the world and received financial support from a record number of corporate sponsors. The 2016 FightSMA Annual Research Conference was held April 7-9 in Alexandria, Virginia.

The FightSMA conference was also the event where the most recent awardees of the Emerging Investigator Awards (EIA) competition were recognized for their achievements. The first EIA competition was in 2012, hosted by FightSMA and the Gwendolyn Strong Foundation. Overall, the program has already contributed more than $650,000 in research funding for all EIA awardees.

“FightSMA’s annual research meeting and the Emerging Investigator Award program support novel research projects and provide a forum to rigorously challenge these new concepts,” said past EIA winner Dr. Barrington G. Burnett of the Uniformed Services University of the Health Sciences in Bethesda, Maryland, in a press release.

“FightSMA’s annual research meeting is an extraordinary opportunity for the best and the brightest in the SMA research community to come together for candid, open discussion,” said FightSMA Research Director Chris Lorson, of the University of Missouri. “It provides an exciting forum not just for sharing data, but also for charting the path ahead.”

Cambridge, Massachusetts-based Biogen was the conference’s presenting sponsor, as the biotech’s main focus is researching potential treatments for neurodegenerative diseases. Other sponsors for the event included Avexis; Hill-Rom; PTC Therapeutics; Genentech; Hoffmann-LaRoche; LabCorp; Ionis Pharmaceuticals; Cytokinetics; Sarepta Therapeutics; the Santa Barbara, California-based Gwendolyn Strong Foundation; and RaNA Therapeutics.

Besides the sponsoring organizations, additional organizations participated, including the Children’s Hospital of Eastern Ontario; Cold Spring Harbor Laboratory; Columbia University; Health Resources and Services Administration, Division of Services for Children with Special Needs; Johns Hopkins; Massachusetts General Hospital, Center for Human Genetics Research; Medical College of Wisconsin; National Human Genome Research Institute, NIH; National Institute of Neurological Disorders and Stroke, NIH; National Institute of Child Health and Human Development, NIH; Nationwide Children’s Hospital; Northwestern University and Lurie Children’s Hospital; Novartis Institutes of Biomedical Research; Ottawa Hospital Research Institute; SMA Foundation; The Ohio State University; Uniformed Services, University of the Health Sciences; University of Edinburgh; and University of Missouri.

“We are thrilled with the record level of support that we are seeing for this year’s research conference,” said FightSMA co-founder Martha Slay. “It is a reflection of how far the SMA research field has advanced over these many years to see the deepening commitment of the biotechnology industry.”