Team
Founders
The Take Down Duchenne foundation was created to support and expand the scientific and advocacy work of Kana Muraki and Larry Miao on behalf of their family and others with dystrophin mutations.
Past projects, supported by expert scientists and clinicians, include new disease model creation, novel antisense treatment design, and cardiac care policy work.
Kana Muraki
Founder and President, Take Down Duchenne
Since her son’s diagnosis with Duchenne muscular dystrophy, Kana has dedicated her time to advocating for patients and families living with Duchenne and advancing initiatives that can meaningfully improve their lives. Through Take Down Duchenne (TDD), she works at the intersection of patient advocacy, research, and therapeutic development. Her work includes advancing initiatives in cardiac care and carrier care, collaborating with scientists on new and emerging therapies and research tools, and helping bring the perspectives and needs of patients and families into the research and drug-development process. Kana previously worked in marketing and venture capital.
Larry Miao
Founder and Treasurer, Take Down Duchenne
Larry Miao is a co-founder of the private equity firm Olympus Capital Asia. He serves on the Stanford University Cancer Institute Advisory Board and has served on the Board of the California Academy of Sciences and the advisory board to the President of Princeton University. He resides in San Francisco and the proud parent of Madeline, Katherine and Sammy
Mary Lee MacKichan, PhD
Executive Director, Take Down Duchenne
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Board & Advisors
Network of experts
Take Down Duchenne has long-standing relationships with independent scientists and experts in Duchenne muscular dystrophy, cardiac care, and policy.
Parent Project Muscular Dystrophy
Strong relationship with PPMD and informal contributions to their public policy and scientific initiatives.
Kanneboyina Nagaraju, PhD, Dean, School of Pharmacy and Pharmaceutical Science, SUNY, Binghamton
Nagaraju is an expert on translational research in autoimmune and genetic muscle diseases, notably Duchenne muscular dystrophy. The author of over 200 refereed publications, he pioneered the generation of novel inducible transgenic and knockout mouse models for muscle and neuromuscular diseases. Early drug screening efforts in his laboratory in collaboration with Eric Hoffman and John McCall led to the identification of Vamorolone (AGAMREE), a potent anti-inflammatory, membrane-stabilizing dissociated glucocorticoid, now approved for DMD patients AGAMREE. Nagaraju has led international efforts to define reliable and sensitive tests for drug efficacy in mouse models of neuromuscular diseases and directed a state-of-the-art preclinical drug-testing facility dedicated to neuromuscular disorders. Nagaraju has received several million in grant support from the NIH and several foundations. He currently serves on multiple medical and scientific advisory boards and on the editorial boards of several specialty journals. He is a founding core preclinical expert of the TREAT-NMD Advisory Committee for Therapeutics and serves on the Board of Trustees at TREAT -NMD Alliance Ltd.
Dr. Nagaraju trained in veterinary science and completed a PhD in immunology before joining the medical school faculties of Johns Hopkins and later George Washington University. He is a past director of the Research Center of Genetic Medicine at Children’s Nationwide Hospital. He moved to SUNY Binghamton to build a new school of pharmacy. He was named Dean in 2022 and granted a SUNY Distinguished Professorship in 2024.
Hubert Kim, MD PhD, Professor, University of California, San Franciso and Chief of Surgical Service at San Francisco VA Medical Cent
Dr. Hubert Kim has a broad background in orthopaedic surgery research and orthopaedic basic science with specific training and expertise in cellular and tissue structure-function analyses of translational animal models. Dr. Kim established the San Francisco VA Laboratory for Translation Orthopaedic Research in 1998 and serves as its Director. His research career has focused broadly on injury, repair, and regeneration of the musculoskeletal system. He also has a long-standing interest in the role of telomerase in musculoskeletal stem cells and has published in this domain as senior author. More recently, Dr. Kim has worked specifically on muscle stem cells and their role in muscle regeneration, as well as a rare muscle disease, Duchenne Muscular Dystrophy. The latter effort encompasses creation of novel tools, including a new Duchenne/Becker muscular dystrophy mouse model to expand mouse model options to support N-terminal mutation and patient-specific research. As PI and Co-PI on multiple VA MERIT, DOD, NIH, and other institutional awards, Dr. Kim has successfully directed and administered a broad spectrum of projects ranging from clinical trials to mechanistic basic science research.
Dr. Kim is a graduate of Stanford University and Stanford University School of Medicine, where he earned both medical and doctoral degrees. He completed a residency in orthopedic surgery at UCSF before joining the faculty in 1998.
Roger Wu, Title
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Steven, Title
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Toshifumi Yokota, PhD, Professor and Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair, University of Alberta
Dr. Yokota is an expert in therapeutic antisense oligonucleotide design and testing for neuromuscular disease, with an extensive track record in Duchenne. His overarching research goal is to improve the lives of people living with devastating genetic disorders using state-of-the-art technologies. Dr. Yokota was the first to demonstrate antisense-based restoration of dystrophin expression and improve muscle strength in a severe animal model of Duchenne muscular dystrophy (DMD). This study directly led to a collaboration with a Japanese pharmaceutical company Nippon Shinyaku and the development of viltolarsen, an antisense oligonucleotide drug to treat DMD. Viltolarsen was approved by the FDA in 2020 based on international trial results showing it restored dystrophin and improved muscle function in clinical trials.
Dr. Yokota's research team designed and tested synthetic DNA-like molecules called gapmers that interfere with the production of a toxic protein that destroys the muscles in facioscapulohumeral muscular dystrophy (FSHD), the third most common form of muscular dystrophy. They demonstrated that the treatment knocked down more than 99 percent of the toxic gene products called DUX4 in patient-derived cells accompanied by morphological and functional improvement. D
Yokota obtained a PhD degree from the University of Tokyo and moved to Imperial College London for postdoctoral training. He has published more than 100 papers, edited 2 books, and served as a board member for numerous journals and organizations.
Rachel Shay, parent
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Pat Furlong, Founding President and CEO Parent Project Muscular Dystrophy
A trained nurse, Pat Furlong was inspired to found the Parent Project Muscular Dystrophy (PPMD) in 1994 by her two sons, who have since lost their battle with Duchenne. PPMD honors their memory through research, advocacy, education, and compassion to improve the lives of all individuals affected by Duchenne. Pat is a member of multiple committees and boards, including the National Health Council, CTTI (Clinical Trials Transformation Initiative) and the data safety monitoring board for both the Rare Diseases Clinical Research Network and Cooperative International Neuromuscular Research Group. and speaks regularly on behalf of the Duchenne community in regulatory, scientific and clinical settings, sharing her broad view of the Duchenne landscape and intimate knowledge of patient needs.
