Speakers

Keynote Speakers

Jim Wilson, MD, PhD

University of Pennsylvania

James M. Wilson, MD, PhD, is a Professor in the Perelman School of Medicine at the University of Pennsylvania where he has led an effort to develop the field of gene therapy. His research career spanning over 40 years has focused on rare diseases and ways to treat them by gene therapy. Dr. Wilson has published over 600 papers and is named on over 200 patents worldwide. The Wilson lab identified a new type of vector based on novel isolates of adeno-associated viruses which have become best in class for gene therapy. At this time, there are more than 80 preclinical and over 40 clinical programs that use Wilson’s AAV vectors. More recently Dr. Wilson’s laboratory has focused on improved vectors for gene therapy and clinical applications of genome editing and mRNA therapy.

Arthur T Suckow, PhD

DTx Pharma

 

 

Dr. Suckow received his BS from the University of Delaware and his PhD from University of California, San Diego.  He has worked for Johnson & Johnson, AstraZeneca and Regulus Therapeutics. Dr. Suckow’s extensive experience in fatty acid pharmacology includes advancing small molecules targeting fatty acids and fatty-acid conjugated peptides into early clinical development.  Dr. Suckow has received numerous awards including a Beckman Fellowship, NSF graduate research fellowship, a BIOCOM catalyst award and many others.

Guest Speakers

Robert "Bob" Baloh, MD, PhD

Roche Pharmaceuticals

Dr. Baloh received a MD-PhD degree from Washington University in 2001, and completed neurology residency at Massachusetts General/Brigham and Women’s Hospitals in Boston. He returned to Washington University in 2005 for a fellowship in neuromuscular diseases and then developed a laboratory research program focused on understanding the molecular mechanisms of neurodegenerative diseases including motor neuron diseases and inherited neuropathies. In 2011 Dr. Baloh moved to Cedars-Sinai Medical Center to head the newly formed Division of Neuromuscular Medicine, and developed specialized programs in ALS/frontotemporal dementia, Charcot-Marie-Tooth disease, and muscular dystrophy with a focus on multidisciplinary patient care and clinical research. In 2020 he joined Roche Pharmaceuticals as VP and Global Head of Research in Neuroscience and Rare Diseases in Basel Switzerland.  

Robert Burgess, PhD

The Jackson Laboratory

Dr. Burgess received his B.S. in Biochemistry from Michigan State University (1990) and his Ph.D. in Neuroscience from Stanford University (1996).  After a postdoctoral fellowship at Washington University, St. Louis, Dr. Burgess took a faculty position at The Jackson Laboratory in Bar Harbor in 2001 and is now a full Professor.  Research in the Burgess lab seeks to understand the molecular mechanisms of synapse formation and maintenance at two sites in the nervous system: the peripheral neuromuscular junction and the retina. In all of these studies, we are addressing basic molecular mechanisms, but these basic mechanisms have relevance to human neuromuscular and neurodevelopmental disorders. The lab is now heavily invested in developing and studying precision models of inherited peripheral neuropathies (Charcot-Marie-Tooth disease).  We are increasingly using the mouse models we have generated to test therapeutic strategies in preclinical studies, especially related to gene therapy approaches, with the goal of translating these findings to patients.  Dr. Burgess has also assumed a number of leadership roles both institutionally and nationally.  Of particular relevance, he is on the scientific advisory boards of the Charcot-Marie-Tooth Association, the Hereditary Neuropathy Foundation and the Talia Duff Foundation, and he was the Chair of the NIH study section for Cellular and Molecular Biology of Neurodegeneration until 2020.  Internally, he is the director for the cooperating Ph.D. program in Neuroscience with Tufts University.

Bruce Carter, PhD

Vanderbilt University

Dr. Carter is Professor of Biochemistry and Associate Director of the Brain Institute at Vanderbilt University. After completing his bachelor’s degree at Alma College, he obtained his PhD in Biological Chemistry from the University of Michigan. He did postdoctoral training with Yves Barde at the Max Plank Institute in Munich, Germany and with Moses Chao at Cornell Medical School.

In 1997 Dr. Carter joined the Department of Biochemistry at Vanderbilt University School of Medicine. His research focuses on the mechanisms by which neurotrophins regulate cell death in the developing peripheral nervous system as well as the mechanisms underlying Schwann cell development and dysregulation, including in models of CMT.

Maurizio D'Antonio, PhD

Ospedale San Raffaele

Dr. Maurizio D’Antonio is Group Leader of the Biology of Myelin Unit in the Division of Cell Biology and Genetics at the San Raffaele Hospital and Research Institute. He is an internationally recognized expert in CMT1B and the role of the unfolded protein response in CMT. For his innovative research, Dr. D’Antonio has won multiple awards, including a Medical Research Council UK fellowship, the Peripheral Nerve Society Arthur K. Asbury Award, and two Young Investigator Awards from the Italian Ministry of Health. Prior to joining the faculty at San Raffaele, he earned his PhD in Cellular and Molecular Biology at University College London.

Keith Fargo, PhD

CMT Research Foundation

Keith joined the CMT Research Foundation after working for 23 years in the research field, including clinical trials, academia, and nonprofit experience. Most recently, Keith worked for seven years at the Alzheimer’s Association, where he led efforts to grow the largest international research conference and scientific society related to dementia science, along with growing engagement in clinical trials. He also led the team of researchers who annually produced Alzheimer’s Disease Facts and Figures, the definitive source of information related to Alzheimer’s incidence and prevalence, mortality and morbidity, and use and costs of care. Keith also brings expertise in public communication of research, making science accessible to people of all backgrounds.

 

 After earning his PhD in neuroscience at Indiana University, Keith worked in peripheral nerve science at Edward Hines, Jr, VA Hospital in the Rehabilitation Research & Development Service, and at Loyola University Chicago—Stritch School of Medicine in the Department of Molecular Pharmacology and Therapeutics.

 

Keith joined the CMTRF in order to help catalyze scientific progress in this exciting field, and will bring his experience to bear on our critical research mission.  He is based in the Chicago area with his wife Susan and 3 children, where they enjoy family life, reading, and Formula 1 racing.

Shawna Feely, MS, CGC

University of Iowa

Shawna Feely is a genetic counselor with a specialty Charcot Marie Tooth disease. She is also the Project Manager for the Inherited Neuropathies Consortium (INC) and helps to coordinate the ~20 sites that are enrolling patients with CMT into studies across the INC.  She was previously an Assistant Professor at Wayne State University (WSU) where she helped to develop a multidisciplinary Center of Excellence for CMT along with providing genetic counseling services to patients in the Department of Neurology. She was recruited the University of Iowa in 2012 to help build a similar CMT program. Shawna is a member of the CMT Association (CMTA) medical advisory board.  She continues to specialize in neurogenetic diseases including CMT and has many publications related to this with her particular area of research involving CMT type 2A (CMT2A) and the identification of novel or new forms of CMT. 

Amanda Haidet-Phillips, PhD

Novartis Gene Therapies

Dr. Haidet-Phillips serves as part of Novartis Gene Therapies team to help transition gene therapy products from the pre-clinical proof of concept stage in R&D to first-in human trials. This includes ensuring trial readiness, capturing stakeholder engagement, and facilitating the design and IND approval for potentially label-enabling trials. Prior to Novartis Gene Therapies, Dr. Haidet-Phillips served as Scientific Portfolio Director for the Muscular Dystrophy Association where she led the strategic management of research grants and investments focused on Charcot-Marie-Tooth Disease, Amyotrophic Lateral Sclerosis, Spinal Muscular Atrophy, and Myasthenia Gravis. Dr. Haidet-Phillips completed a post-doctoral fellowship at Johns Hopkins University seeking to understand the causes of ALS and develop new therapeutic approaches for the disease. She received her Ph.D from The Ohio State University where she helped to develop gene therapies for neuromuscular disease.

David Herrmann, MBBCh

University of Rochester

Dr. Herrmann is Unit Chief of the Neuromuscular Unit & Director of the Peripheral Neuropathy Clinics and Cutaneous Innervation Laboratory at the University of Rochester. Clinically his interests are in the diagnosis and care of patients with various types of peripheral neuropathy. He has a particular interest in inherited neuropathies (Charcot Marie Tooth neuropathy and related disorders). Dr. Herrmann additionally established one of the first cutaneous innervation laboratories in the USA at University of Rochester in 2000, for diagnosis of small fiber neuropathy. Dr. Herrmann’s current research interests include inherited neuropathies and he is Principal Investigator in Rochester for the NIH sponsored Inherited Neuropathy Consortium Rare Disease Clinical Research Center. Dr. Herrmann focuses on new gene identification for inherited neuropathies, development of novel neuropathy outcome measures and biomarkers and experimental therapeutics.

Erika L. F. Holzbaur, PhD

University of Pennsylvania

Erika L. F. Holzbaur is the William Maul Measey Professor of Physiology at the University of Pennsylvania Perelman School of Medicine.  She received her B.S. with High Honors in Chemistry and History from the College of William and Mary and her PhD in Biochemistry from Penn State. Holzbaur joined the faculty at Penn in 1992, where she is now an endowed professor in the Department of Physiology.  The Holzbaur lab studies the dynamics of organelle motility along the cellular cytoskeleton. This motility is required to drive the active transport of vesicles and organelles over distances of up to 1 meter along the axons and dendrites of neurons. The lab also investigates cellular mechanisms leading to neurodegeneration, focusing on the dynamics of autophagy and mitophagy in neurons, pathways which are critical to maintain neuronal homeostasis. Dr. Holzbaur has received a Porter Fellowship, the NINDS Javits Award, the Stanley N. Cohen Biomedical Research Award, the Jane M. Glick Graduate Student Teaching Award, and is a Lifetime Fellow and President-Elect of the American Society for Cell Biology.  More than 40 postdocs and students have trained in her lab, many now on faculties at major research institutes. 

Sung-Chul Jung, MD, PhD

Ewha Womans University

Prof. Sung-Chul Jung is currently a professor of Biochemistry at College of Medicine, Ewha Womans University in Seoul, Korea. In addition, he serves as an associate dean for research of College of Medicine and the Director of Medical Research Institute at Ewha Womans University. He received an MD degree from College of Medicine, Hanyang University in 1989, and got a PhD degree from Hanyang University, Seoul, Korea in 1997. His main research subjects are inherited metabolic diseases and inherited peripheral neuropathies. He is interested in development of novel therapeutic strategies such as enzyme replacement, cell and gene therapies for treatment of genetic diseases. He has published over 100 peer-reviewed articles. In present, Prof. Jung and his colleagues are trying to develop cell therapy for patients with peripheral nerve diseases including Charcot-Marie-Tooth disease. For developing cell therapy for clinical use, they are collaborating with Cellatoz Therapeutics, Inc, an innovative cell therapy company in Korea. 

Kleopas Kleopa, MD

Cyprus Institute of Neurology & Genetics

Prof. Kleopas A. Kleopa holds a medical and a doctoral degree from the University of Wuerzburg, Germany and has trained as a neurologist at Drexel University in Philadelphia, followed by a fellowship in neuromuscular disorders and electromyography at the University of Pennsylvania. He is currently a consultant neurologist at the Cyprus Institute of Neurology and Genetics, where he coordinates the Center for Neuromuscular Disorders. He is also Head of the Neuroscience Department and Professor at the Cyprus School of Molecular Medicine coordinating the Neuroscience Graduate Program and has mentored several MSc and PhD students, as well as postdoctoral fellows. His research group has made significant contributions to the understanding of disease mechanisms and to the development of cell-targeted gene therapies for inherited demyelinating neuropathies and leukodystrophies. He has received many prestigious National and International Awards for his academic achievements including the 2015 European Academy of Neurology Investigator Award for his research on gene therapy for inherited neuropathies and the 2017 National Distinguished Researcher Award from the Research Promotion Foundation of Cyprus. He is an elected Board Member of the Charcot-Marie-Tooth and Related Disorders (CMTR) Consortium and a Member of the Scientific Advisory Board of the CMT Association.

Bryan Lafitte, PhD

DTx Pharma

Chief Scientific Officer of DTx Pharma leveraging 20 years’ experience in biotech and major pharmaceutical companies to help move our science to the next level. Scientist of the Year and VIVA Award winner for 2016 at Novartis. Previously the Director of Regenerative Medicine, Head of Type 1 Diabetes Program and Co-Leader of the Molecular Targeting Initiative at the Genomics Institute of the Novartis Research Foundation. Initiated and led FXR agonist project at Novartis from initial concept to multiple positive Phase 2 clinical trials; developed tropifexor, the cornerstone of Novartis Hepatology, and backup compound nidufexor. Extensive experience in the evaluation of therapeutic targets, drug development, and delivery of high-quality clinical candidates in multiple disease areas, including endocrinology, hepatology and oncology. Former leadership team member founding new companies at Inception Therapeutics.

Kelly Langert, PhD

Loyola University Chicago

Kelly Langert, Ph.D. is an Assistant Professor of Molecular Pharmacology and Neuroscience at Loyola University Chicago’s Stritch School of Medicine and a Research Health Scientist at Edward Hines, Jr., VA Hospital. She studied neurobiology and psychology at the University of Wisconsin- Madison and received her Ph.D. in Neuroscience from Loyola University Chicago before completing postdoctoral training at Illinois Institute of Technology in the Department of Biomedical Engineering. In 2019, Dr. Langert accepted her current tenure-track faculty position and began her independent laboratory focused on targeted drug delivery for inflammatory peripheral neuropathies. Research in the Langert Lab focuses on bringing together strategies based in biomaterials and in neuroscience to advance novel, mechanism-based treatments for disease. Dr. Langert is the recipient of a the ASN Neuro award (2012), VA CDA-I award (2014), and VA CDA-II award (2017), and she served on the executive council of the Chicago Chapter of the Society for Neuroscience from 2017-2021. 

Gabsang Lee, DVM, PhD

Johns Hopkins University

Dr. Lee obtained his bachelor’s, doctoral and doctor of veterinary medicine degrees in Veterinary Medicine at the Seoul National University, South Korea. After his post-doctoral training at Sloan Kettering Institute, New York, he joined the faculty of Johns Hopkins as Assistant Professor in the Department of Neurology and Institute for Cell Engineering (ICE), where he has been promoted to Associate Professor. Dr. Lee has a strong background in modelling human genetic disorders by using reprogramming technology, including induced pluripotent stem cells (iPSCs). He has dedicated his career to establishing a human iPSC-based model system to study peripheral nervous system disorders.

The international scientific community recognizes and values Dr. Lee’s knowledge, as exemplified by the many awards he has received including the Robertson Investigator Award from the New York Stem Cell Foundation (one of the largest awards from private foundations), and his active participation in journal peer review activities and domestic/international research grant programs in the area of stem cell research, neurodegenerative diseases, and high throughput screening. He has been invited to speak at more than 50 conferences in the United States and international locations. 

Cat Lutz, PhD, MBA

The Jackson Laboratory

Dr. Lutz is Director of the Mouse Repository and the Rare and Orphan Disease Center at The Jackson Laboratory.  She has fiscal and managerial oversight of a growing collection of more than 8,500 unique strains, including over 1,700 live colonies for distribution to the scientific community.   As part of the Mouse Repository program, Dr. Lutz is the Principal investigator on a number of NIH sponsored resource grants, including the Mutant Mouse Regional and Research Center at JAX, The SMSR grant to support recombinant inbred and Chromosome substitution panels, as well as the NICHD Cytogenetic Resource to support Down Syndrome related strains and research.   Dr. Lutz also serves as the Director of In Vivo Pharmacology and Efficacy Testing Program in Bar Harbor, which interfaces with biotechnology and pharmaceutical companies to pursue novel therapeutics across a variety of therapeutic areas.  A neuroscientist by training, Dr. Lutz conducts research in neurodegenerative diseases, including Spinal Muscular Atrophy (SMA), Friedreich’s ataxia, Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Lobe Dementia (FTD).

Liliane Massade, PhD

INSERM

Dr. Liliane Massade, PhD, senior researcher at the Centre National de Recherche Scientifique (CNRS) is the head of “The “Targeted therapy for peripheral neuropathies” team (https://dhns-inserm.com/team3/) of the INSERM UMR 1195. She has as expertise in cellular and molecular biology and in the pharmacology of siRNA. Her research goes from basic to translational research and involves close collaborations between clinicians and researchers with research programs structured around neuroprotection, neuroregeneration and myelin repair for peripheral neuropathies with unmet medical needs. She published 71 articles (e.g. Cancer Res., Oncogene, J Control Release, PNAS, Comm. Biology,) and has 2 patents.

Brett McCray, MD, PhD

Johns Hopkins University

Dr. McCray is an Assistant Professor in Neurology at Johns Hopkins. He is a physician-scientist with a clinical and research focus on Charcot-Marie-Tooth (CMT) disease and other forms of peripheral neuropathy. He currently serves as the co-director of the Johns Hopkins CMT clinic and the Johns Hopkins site principal investigator for the Inherited Neuropathy Consortium genetics and natural history CMT study. In addition to caring for patients with CMT and other neuromuscular diseases, he also runs a basic science lab focused on the pathogenesis of inherited forms of peripheral neuropathy with the goal of identifying common pathogenic mechanisms and novel broadly applicable therapeutic targets. His current work is focused on understanding the pathogenesis of CMT type 2C, which is caused by mutations in the calcium-permeable cation channel TRPV4 (transient receptor potential vanilloid 4). He studies animal models of this disease, and he has also established a TRPV4 neuropathy patient registry to better define the natural history of the condition and to work towards a future clinical trial. 

Jeffrey Milbrandt, MD, PhD

Washington University in St. Louis

Jeffrey Milbrandt is the James S. McDonnell Professor and Head of Genetics, Executive Director of the McDonnell Genome Institute, Co-Director of the Needleman Center for Neurometabolism and Axonal Therapeutics, Professor of Pathology & Immunology, Medicine and Neurology, and a member of the Hope Center for Neurological Disorders.

His research laboratory uses genomic and metabolic approaches to understand the process by which unhealthy or injured axons degenerate, an early pathologic event that underlies most neurodegenerative diseases and peripheral neuropathies. His lab has made major discoveries including identification and characterization of the GDNF neurotrophic factor family, and identifying the molecular link between metabolism, NAD and axon health. Most recently, in a joint effort with the DiAntonio lab, his lab found that SARM1, a crucial component of the axon degeneration pathway, is an enzyme that breaks down NAD and is a promising drug target for treatment for degenerative disorders. Together, Milbrandt and Aaron DiAntonio, MD, PhD, are founding co-Directors of the Needleman Center for Neurometabolism and Axonal Therapeutics, which seeks to understand the influence of metabolism on neurodegenerative disease in order to identify new treatments for these disorders. They are also the scientific co-founders of Disarm Therapeutics, a company whose goal is to identify SARM1 inhibitors for treatment of neurodegenerative conditions and which was recently purchased by Eli Lilly and Company. He has co-authored over 250 publications and holds more than 25 patents on neurotrophic factors and axonal protective agents and their use in treating neurological disorders.

William Motley, MD, DPhil

Flare Therapeutics

Dr. Motley is the director of translational medicine at Flare Therapeutics.  Priori to joining Flare, Dr. Motley was a senior associate at Third Rock Ventures, where he worked with academic researchers to launch and build life science companies. He received his bachelor’s degree from Middlebury College, his medical degree from the University of Pennsylvania School of Medicine, and his doctorate at the National Institutes of Health and the University of Oxford, where he was a Marshall Scholar.

He completed a neurology residency at the Johns Hopkins University School of Medicine. His doctoral and post-doctoral research experiences focused on identifying new causes of CMT, and characterizing pathological mechanisms of the disease.

Lukas Neukomm, PhD

University of Lausanne

Lukas Neukomm received his MSc at ETH Zürich, where he studied cellular energetics in Prof. Dr. Theo Walliman’s laboratory. He then moved to the University of Zürich, where he completed his PhD in Molecular Biology under the supervision of Prof. Dr. Michael Hengartner. There, he studied how sick, unhealthy cells are either tolerated, or removed by the surrounding tissue in the roundworm C. elegans. For his postdoctoral training, he moved to the University of Massachusetts Medical School, where he focused on axon degeneration in the fruit fly D. melanogaster, mentored by Prof. Dr. Marc Freeman at the Neurobiology Department/Howard Hughes Medical Institute. He was recently awarded with a Swiss National Science Foundation (SNSF) Assistant Professor position and started his own lab in June 2018.

Susan Ruediger

CMT Research Foundation

Susan Ruediger was diagnosed with CMT1A when she was 18 months old and can trace the genetic disease back five generations in her mother’s family. After the birth of her first child, Susan started a local CMT support group to get more information and resources for CMT. Since 2007, Susan has been deeply engaged with the patient and research communities, building connections to fund efficient and effective research leading toward drug development. Susan is driven to deliver treatments to all people living with CMT. 

Susan brings her strategic planning, operational, communications, fundraising and non-profit management experience to the CMT Research Foundation. As a member of the BIO Patient Advocacy Committee, Susan built a robust network of experts in drug development and patient advocacy, and continues to be sought after by peers in patient-led drug discovery and as an expert in CMT. She shares her knowledge of CMT at Georgia Tech, Emory University, Hanger, Inc., The American Orthotic and Prosthetic Association, and BIO. She is a former board member of the Junior League of Atlanta. Susan holds a Bachelor of Arts from Clemson University.

Zarife Sahenk, MD, PhD

Nationwide Children’s Hospital

Dr. Zarife Sahenk is a professor of Pediatrics and Neurology and the Director of Experimental and Clinical Neuromuscular Laboratories at Abigail Wexner Research Institute at Nationwide Children’s Hospital. Her laboratory plays an integral part in the preclinical studies and the gene therapy clinical trials that take place in the Center for Gene Therapy at Nationwide Children’s Hospital. A primary goal of her research program is to gain a detailed understanding of the anatomical and molecular basis of nerve degeneration and impaired regeneration in hereditary peripheral neuropathies. The role of neurotrophin-3 (NT-3) in impaired nerve and muscle function in animal models of Charcot-Marie-Tooth (CMT) subtypes is currently being investigated and gene therapy paradigms are being developed. In addition, her laboratory works on projects involving the development of therapeutics and novel methods to deliver therapeutic genes more efficiently to the peripheral nerves. The results of Dr. Sahenk’s research in CMT as well as other neuromuscular diseases have been published in more than 100 peer-reviewed articles and her most recent contributions on the topic of CMT include articles in Gene Therapy,  Brain Research and Brain and Behavior.

Oranee Sanmaneechai, MD

Siriraj Hospital Mahidol University

Oranee Sanmaneechai is an associate professor in pediatric neurology in the largest university hospital in Thailand. She has extensive experience in pediatric neuromuscular fields including CMT, SMA and DMD. Her interest in pediatric CMT grew out of her training with Dr. Mike Shy. Her recent research includes developing and validating CMT infant scale and phenotype in children with CMT. She also established a multidisciplinary clinic for neuromuscular diseases in Thailand. She also conducts many clinical trials for children with DMD and SMA. In addition to her research and clinical work, she is also associate dean for research and innovation.

Mario Saporta, MD

University of Miami

Dr. Saporta is a clinician-scientist and neuromuscular specialist with over 15 years of experience in genetic neuropathy research. He is an Associate Professor of Neurology and Human Genetics and the medical director of the University of Miami CMT Center of Excellence. Dr. Saporta conducts both clinical and translational research in CMT. He is the North America Principal Investigator for the Pharnext Premier trial and he leads a research lab focused on developing and applying human cellular models of CMT for drug discovery and genetic therapy validation.

Eroboghene Ubogu, MD

University of Alabama at Birmingham

Dr. Ubogo is a Professor of Neurology and Neurobiology at the University of Alabama at Birmingham (UAB) and the director of the division of Neuromuscular Disease since September 2013. Prior to joining UAB, Dr. Ubogu completed medical school at Imperial College London, an internship at the Cleveland Clinic, an adult neurology residency at the University Hospitals-Case Medical Center, and a fellowship in Clinical Neurophysiology/Neuromuscular Disorders at Emory University, and began his faculty career at Baylor College of Medicine. At UAB, Dr. Ubogu directs the Neuromuscular Immunopathology Research Laboratory (NIRL), a National Institutes of Health-funded research laboratory that published the first comprehensive normal human BNB transcriptome using purified passaged primary human endoneurial endothelial cells and laser capture microdissected endoneurial microvessels from histologically normal cryopreserved adult sural nerve biopsies.

Dr. Ubogu has numerous academic and professional awards, over 120 peer-reviewed publications, written/edited 2 books, written over 50 book chapters and given over 70 scientific presentations and invited lectures. Dr. Ubogu is also board-certified in Neurology, Clinical Neurophysiology, Electrodiagnostic Medicine, Neuromuscular Medicine and Clinical Neuromuscular Pathology

Ludo Van Den Bosch, PhD

VIB, KU Leuven

Our research focuses on the cause, mechanism and treatment options for neurodegenerative diseases. In particular, we focus on amyotrophic lateral sclerosis (ALS, Lou Gehrig’s disease) and its relation to frontotemporal dementia, as well as on Charcot-Marie-Tooth disease (CMT) and related neuropathies. To this end, we are involved in genetic studies, study in vitro models (e.g. iPSC-derived neurons and non-neuronal cells) as well as fruit fly, zebrafish, mouse and rat models, and have an intensive collaboration with the hospital. Our aim is to translate results from these genetic, in vitro and animal studies into new treatment strategies. In order to do so, I recently established a spin-off company (Augustine Therapeutics) to develop a new therapy for CMT based on our observations that inhibitors of histone deacetylase 6 (HDAC6) are able to counteract the problems in different CMT models, as well as in models of induced neuropathies.

Donald S. Wood, PhD

Muscular Dystrophy Association (MDA)

Donald S. Wood, PhD, is president and chief executive officer of the Muscular Dystrophy Association (MDA). He has been involved with MDA for nearly 40 years, most recently as vice chairman of the board of directors. Most notably, Dr. Wood launched and managed MDA’s Task Force on Genetics that led to the discovery of the gene that causes Duchenne muscular dystrophy (DMD).

Dr. Wood has more than four decades of experience leading both nonprofit and for-profit institutions. Prior to his current position, he served as vice president of institutional effectiveness at Odessa College where his work developing a Drop Rate Improvement Program is credited with dramatically improving student outcomes. Throughout the 1990s and early 2000s, he served first as an executive and then as president and chief operating officer of IntegraMedAmerica, Inc., a medical services provider. During this time, he also helped create a new genetic-testing company, Odyssey Biomedical, which was later sold to Integrated Genetics, a division of Genzyme.

Dr. Wood began his career in neuromuscular disease research at Columbia Presbyterian Medical Center in New York, where he served as a National Institutes of Health (NIH) postdoctoral research fellow in neurology. He holds a doctorate in physiology from Washington State University.

Grace Zhai, PhD

University of Miami

Research in Dr. Zhai’s lab is focused on understanding the genetic and molecular mechanisms of neural degeneration and protection in the context of both common and rare neurodegenerative diseases. They use a “Drosophila – mammalian tissue culture two-model” systems, to identify genetic components in Drosophila and characterize the cellular mechanisms in mammalian cells.   Dr. Zhai has established and developed a strong, innovative, and well-funded research program that includes three related yet independent research directions.  Each direction is supported by high impact publications and federal funding.

Ruxu Zhang, MD

Third Xiangya Hospital, Central South University


Dr Ruxu Zhang is a Neurologist  at the Third Xiangya Hospital, Central South University in China. she leads the working Group of Inherited Peripheral Neuropathies. Her research focuses on the discovery of novel causes of inherited neuropathies, nature history study and good clinical practices in Charcot-Marie-Tooth disease.