Dr. Gordish-Dressman is an Assistant Professor of Pediatrics and Integrative Systems Biology at George Washington University School of Medicine and Health Sciences and in the Center for Genetic Medicine Research at Children's National Medical Center. Dr. Gordish-Dressman has worked with Dr. Eric Hoffman and served as the primary statistician for the Center for Genetic Medicine Research since 2002. She specializes in the analysis of various types of genetic data including large-scale cohort studies, proteomic data, microarray data and single nucleotide polymorphism data in both candidate gene and GWAS studies. She has served as the statistician for Dr. Kanneboyina Nagaraju’s Pre-clinical Murine Testing Facility at CNMC and continues this work as a consulting statistician for AGADA BioSciences. Since 2010, Dr. Gordish-Dressman has provided statistical support to the Cooperative International Neuromuscular Research Group (CINRG) under the direction of Dr. Cnaan. Here she focused on the analysis of natural history studies and facilitated collaborations with academic and industry partners. Dr. Gordish-Dressman has provided statistical support to a large number of collaborators both within CNMC and at other institutions around the world. She has co-authored 100 scientific publications on a wide variety of topics including, Duchenne muscular dystrophy, cerebral palsy, asthma, necrotizing enterocolitis, biomarkers of disease and drug treatment, obesity and metabolic traits, and muscle strength and fitness.
Dr. Hoffman is a human geneticist and translational researcher focused on neuromuscular disease, and skeletal muscle tissue in health and disease. His most recent efforts focus on drug development and clinical trials in Duchenne muscular dystrophy. He received his PhD in Drosophila molecular genetics from Johns Hopkins University, then carried out a post-doctoral fellowship at Boston Children’s Hospital and Harvard Medical School working on the identification of the DMD gene and protein. His current academic positions are Associate Dean for Research, School of Pharmacy and Pharmaceutical Sciences, Binghamton University – SUNY, where he is building a research program focused on facilitating drug development at the academic/industry interface. In the private sector, he is co-founder and CEO of ReveraGen Biopharma and co-founder and Vice President of AGADA Biosciences. He serves on Scientific Advisory Boards of Foundation to Eradicate Duchenne, CureDuchenne Foundation, C3 Foundation, Save Our Sons Foundation, and Duchenne UK. Research accomplishments include identification of the dystrophin protein, and defining its deficiency in DMD patient muscle, mdx mouse, dog and cat models. His lab identified initial voltage sensitive ion channel mutations in human disease, and genetic causes of recurrent pregnancy loss. He is an inventor on nine patents, and has authored over 500 publications.
Dr. Knowles joined CureDuchenne in 2015 as the Managing Director of CD Ventures and the Vice President of Medical and Scientific Affairs for CureDuchenne. Jak has had a career in healthcare spanning over a decade with the last five years focused in biotech venture capital and finance. Most recently, Jak was as an investment professional in the Boston office of MPM Capital ($2B AUM) where he worked within the BioVentures Asset Management team starting early-stage companies focused on developing curative therapeutics across a wide spectrum of indications. Prior to MPM, Jak worked in New York as a biotechnology equities analyst within the investment bank JMP Securities, and with Burrill & Co., a life sciences VC firm. Jak began his post-graduate business career working in health and life sciences consulting practice of Oliver Wyman where he helped implement value-based transformation of hospital systems and physician practices through re-designing care-models, implementation of electronic clinical decision support, and structuring reimbursement around patient centric care. Jak has a medical degree from Stanford University where he graduated with honors in biomedical research, and a B.S. in biology from Binghamton University. Jak has authored over a dozen publications from research performed at the Memorial Sloan Kettering Cancer Center, Stanford University, and during a NIH post-doctoral surgical research fellowship. Currently, Jak serves on the Board of Directors for Myotherix, RASRx, and Bamboo Therapeutics.