Since 1991, FightSMA has had close relationships with researchers working to find a treatment or cure for Spinal Muscular Atrophy. In the video below, you hear from some of those scientists talking about our organization.
Below are some additional quotes from researchers with whom we've worked.
The Richmond connection to FightSMA has served as a paradigm for cooperation for education and research in this critically important neuromuscular disease.
Dr. Bob Leshner
San Diego School of Medicine
The drug we developed with Ionis Pharmaceuticals, ISIS-SMNRx, is in pivotal phase-3 clinical trials in infants and children, and we are extremely hopeful. For me, it all started with the seed funding that FightSMA gave us in January of 2000, to begin to study why the C to T change in exon 7 of SMN2 causes it to be skipped.
Adrian Krainer, Ph.D.
Cold Spring Harbor Laboratory
Cold Spring Harbor, NY
As a beginning assistant professor of genetics at Columbia University, I was mindful not to come across as too intense. That was before I met Joe Slay. Over a frantic 30-day period in the Fall of 1991, Joe and the wonderful citizens of Richmond raised over $100,000 to purchase an automated DNA sequencer for my lab. An unheard of acquisition for a starting professor, my group ran that machine night and day until we excavated more than 5 million base pairs of DNA that included the gene responsible for SMA. The citizens of Richmond helped change the course of history that fall.
T. Conrad Gilliam, Ph.D.
University of Chicago
The enormous generosity of the Richmond community in supporting the Fight against SMA has had an everlasting impact on the SMA research effort. It has not only supported key research efforts that have led to where we are today (on the verge of our first treatments), but has supported an annual meeting which enables researchers to integrate their efforts, and perhaps most importantly has provided seed money to support the careers of young people in the field.
Dr. Charlotte Sumner
Johns Hopkins School of Medicine
Your support to me for the STOP SMA study was and is invaluable and that study would not have been possible were it not for the support of FightSMA. The whole premise for the community support for the NeuroNext SMA biomarker study and the current NURTURE trial is based on preliminary data that your seed funding provided...I can't even begin to express how grateful I am for the ongoing support to our research community that you and the entire FIghtSMA team have provided.
Dr. Kathy Swoboda
Massachusetts General Hospital
Through your generous contributions I, am excited for the opportunity to currently investigate the pharmacodynamics of a novel small molecule that increases SMN protein. It is my hope that this research will identify a promising candidate treatment for spinal muscular atrophy. All the projects funded by FightSMA over the past 25 years have helped to identify the cause of the disease and the development of targeted research projects to fundamentally improve the life of children with SMA.
Anne Reitz, Ph.D.
Neuroscience, Virology, Cancer Research
I am a recently graduated researcher who is interested in finding a cure for this devastating disease, Spinal Muscular Atrophy. Your organization’s financial assistance has allowed numerous young researchers to be one step closer to their goals and has inspired all of us in the SMA field to help others by giving back to the community.
Erik Y. Osman, Ph.D.
University of Missouri
For nearly 25 years now FightSMA has been at the forefront of SMA patient advocacy, advancing our understanding of the disease and funding innovative therapeutic programs. In the current funding climate, new investigators, like myself, are finding it extremely difficult to maintain our work on SMA. In the form of the annual research meeting and Emerging Investigator Award Program, FightSMA continues to support novel research projects and provide the forum to rigorously challenge these new concepts. The SMA research community owes a debt of gratitude to FightSMA, without whom much of our work would not be possible.
Barrington G. Burnett, Ph.D
Uniformed Services University of the Health Sciences
I want to extend my personal gratitude to the Richmond community for their continuous support for Spinal Muscular Atrophy (SMA) research. You all are huge source of inspiration and a driving force for the fight against SMA. You all are making a huge difference in the lives of SMA patients and their families. I hope that as always, the Richmond community will continue their support in our collective quest to cure SMA.
Faraz Farooq, Ph.D
Children’s Hospital of Eastern Ontario
I have been committed to understanding the basic mechanism of disease in SMA for the past 12 years. Receiving an award from FightSMA has given me the opportunity to expand my research to include translational projects that can directly benefit the SMA community.
Eileen Workman, Ph.D.
Congratulations on 25 years. Your endless dedication towards moving research forward….well, there are no words to truly express how you have impacted SMA research, families and researchers. ‘Thank you’ seems very inadequate.
Monique Lorson, Ph.D.
University of Missouri
Here at the University of Oxford, we are so grateful for the generosity and support of the Richmond SMA community. With your help, we are able to further our research on better understanding metabolic perturbations in SMA and how they impact the day-to-day nutritional care of SMA patients.
With your support, FightSMA has been able to fund valuable resources and also lead the fight in Capitol Hill to bring awareness of SMA and other orphan diseases into the public eye and highlight the urgency for rapid and sweeping clinical progress in pediatric disease research.
As a recipient of an Emerging Investigator award, funds from FightSMA and the Gwendolyn Strong Foundation advanced SMA-based research in my lab for two consecutive years. Not only is this important for the advancement in research, but this award also helps us generate data and recognition which will be instrumental in securing further research funding from additional sources. The prestige associated with this award was also instrumental for me to secure my position in order to be able to start my own research group. The award not only funded SMA-based research, but has also been key for future prospects of SMA research in my laboratory. I have also found the annual FightSMA conference to be one of the most valuable conferences of the year, and I feel very fortunate to be able to network with such a prestigious group of researchers.
Lyndsay Murray, Ph.D.
University of Edinburgh
Chris Lorson and Eric Hahnen were Fellows in my lab at Tufts University and had made the key discovery of the importance of splicing due to a single nucleotide within the SMN2 gene. This explained a lot. We subsequently devised an assay, which FightSMA supported, that has been used by us and copied by others, including pharmaceutical companies.
Elliot Androphy, Ph.D.
FightSMA’s support was instrumental in helping me start up a new laboratory with the goal of better understanding how the neural circuits controlling breathing and movement are affected by SMA. The Emerging Investigator Award I received has allowed me to pursue new ideas that have never been tested before and therefore are not likely to be funded by conservative government funding agencies. I want you to know how much it means to me and other emerging investigators to get the support to pursue exciting new ideas.
Steven Crone, Ph. D.
Cincinnati Children's Hospital Medical Center
I'm amazed by how far the field has advanced through the sheer will of affected families and those that support them. That continued support is crucial to our ongoing mission to better the lives of those affected by SMA. Funding from FightSMA has been essential for our research into factors that affect quality of life for SMA patients.
Kevin Foust, Ph.D.
The Ohio State University
I want to say how important it is to support initiatives that FightSMA has supported, particularly early on, when they are risky. After all, you funded the assay that did get used for developing compounds that alter splicing. And now, gene therapy is in clinical trials using AAV9. FightSMA supported the development of the first gene therapy vectors using EIAV vectors made by Oxford Biomedica. These studies set the path to the development of AAV9. Fight SMA has supported many innovative projects that have led to the foundations of the treatments in clinical trials. The Burghes laboratory deeply appreciates this support which advances SMA research. Research is often less defined, and many surprises occur. FightSMA and the Richmond community supported early research in SMA that often was not as clear as its outcome has now become. This has had major significance laying the foundation for high-throughput screens for molecules that increase SMN.
Arthur Burghes, Ph.D.
The Ohio State University