Speakers

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	Rachael Anatol PhD, OTP Super Office Deputy Director, FDA Rachael Anatol, Ph.D., is Deputy Super Office Director, Office of Therapeutic Products (OTP), Center for Biologics Evaluation and Research (CBER), FDA, and Acting Chief of the Policy and Special Projects Staff. OTP regulates cell and gene therapies, plasma protein therapeutics, xenotransplantation products, human tissues, devices, and combination products. Rachael, together with the Super Office Director, Nicole Verdun, is responsible for all regulatory, policy, and research work conducted in OTP. Between December 2022 and December 2023, Rachael served as Senior Vice President for Science and Regulatory Affairs at the Biotechnology Innovation Organization (BIO). In this role, Rachael led all aspects of BIO’s science and regulatory advocacy function, managed the BIO Board Regulatory Environment Committee, and advised executive and senior-level BIO staff on BIO’s science and regulatory agenda. Prior to joining BIO in December 2022, Rachael spent 16 years at the Food and Drug Administration, where she took on roles of increasing responsibility in the CBER, culminating as Deputy Director of the Office of Tissues and Advanced Therapies (the predecessor office to OTP). Rachael received her Ph.D. in molecular and cell biology from the University of Maryland, College Park, and completed her post-doctoral training at NIH’s National Heart, Lung, and Blood Institute.

	Charlotte Barrett-Weber MPS I Parent Charlotte Barrett-Weber became an MPS I parent in 2019. Her son, Titus, was diagnosed through newborn screening in KY & transplanted at Cincinnati Children’s Hospital at 3 months of age. Charlotte has been a registered nurse for 11 years and is currently practicing part time in a rural hospital. Charlotte is passionate about sharing Titus’ journey through social media and in her local community. Through doing so, it grants her with the opportunity to provide awareness and education on MPS I, and most importantly, to give hope to newly diagnosed families.

	Lisa Berry Cincinnati Children's Hospital Lisa Berry is a genetic counselor with the Rare Genetic Disease Program at Cincinnati Children’s Hospital. She began to work with individuals and families whose lives have been impacted by lysosomal storage disorders in 2008. In addition to providing genetic counseling for families, she is on the Ohio Newborn Screening Advisory Council and is a member of the Board of Directors for the National MPS Society. Her main roles are treatment/care coordination, advocacy and working on clinical trials for various LSDs.

	Barbara Burton M MD Dr. Barbara K. Burton is a Professor of Pediatrics at the Northwestern University Feinberg School of Medicine and an Attending Physician in the Division of Genetics, Birth Defects and Metabolism at the Ann & Robert H. Lurie Children’s Hospital of Chicago. She is Board certified in Pediatrics, Clinical Genetics and Clinical Biochemical Genetics. Her clinical and research interests are focused on inborn errors of metabolism and newborn screening. Dr. Burton is an investigator in numerous natural history studies and clinical trials of new therapies for various metabolic disorders including many for the MPS disorders. She has published over 250 peer-reviewed articles, 50 chapters in books and is an editor of two textbooks. Dr. Burton is active in professional organizations and is a Past-President of the Society for Inherited Metabolic Disorders and the Chicago Pediatric Society. She served for four years as a member of the Secretary’s Advisory Committee on Heritable Disorders in Infants and Children, the federal advisory committee that makes recommendations regarding newborn screening in the US and served for many years as Chairman of the Newborn Screening Advisory Committee of the Illinois Department of Public Health. She is an Emeritus Member of the Board of Directors of the Greater Chicago Area March of Dimes and received a Lifetime Achievement Award from the March of Dimes in 2017. She received the PKU Hero Award from the National PKU Alliance in 2018. She is a member of the Scientific Advisory Board of the National MPS Society and serves on the medical advisory board of a number of other patient advocacy organizations.

	Emma Canepa UCSF Emma Canepa is a Program Manager for clinical trials with the Center for Maternal-Fetal Precision Medicine at UCSF. Emma received her BA in Molecular and Cell Biology with a focus on Immunology from the University of California, Berkeley, and her MS in Health Policy and Law from UCSF/UC Law. Emma’s work in clinical research has focused on rare genetic disorders and has spanned fetal, pediatric, and adult studies from both industry- and investigator-initiated protocols.

	Caitlin Cooney MS, CGC Caitlin Cooney is a board certified and licensed genetic counselor with experience working in clinics, laboratories, and health-tech startups. She received her master’s degree in Genetic Counseling from Sarah Lawrence College in 2014 and started her career as a clinical genetic counselor with two large NYC hospitals. She later focused on tele-counseling for various genetic tests and supported a large team of laboratory genetic counselors. Caitlin is now genetic counselor with Geisinger and her role will be focused on supporting GenomeConnect.

	Kate Delaney Senior Director, Patient Advocacy at BioMarin Pharmaceutical, Inc.

	Mike Denne PharmD, Takeda Mike is a Pharm.D., trained as a clinical pharmacist, and joined the pharmaceutical industry in 2005 specializing in Medical Affairs. He as been focused on Rare and Orphan Disease for the past fifteen years. He is particularly passionate about improving outcomes in these diseases, from diagnosis through aging, by advancing research and partnering in the education of healthcare providers caring for patients with rare diseases.

	Patricia Dickson MD, Washington University School of Medicine Dr. Patricia Dickson received her medical degree from Columbia University and an AB in Classics from the University of Chicago. She is a diplomate in General Pediatrics and Medical Genetics (both Clinical Genetics and Medical Biochemical Genetics). Since 2003, she has researched brain-directed enzyme replacement therapy for MPS. Her research efforts have encompassed MPS I, IIIB, and IIID, with past and ongoing research supported by the National Institutes of Health, the Food and Drug Administration, private foundations, and industry. She has co-organized national workshops on the challenges in developing therapies for the brain in inborn errors of metabolism and on central nervous system (CNS) drug delivery. Dr. Dickson is a member of and past Chair of the National MPS Society Scientific Advisory Board and has also served on several advisory boards and data and safety monitoring boards for pharmaceutical companies who are designing and conducting clinical trials of CNS-directed therapy for rare diseases.

	Julie Eisengart PhD, University of Minnesota Julie Eisengart is an Associate Professor of Pediatrics at the University of Minnesota Medical School and the Director of the Neurodevelopmental Program in Rare Disease. As a pediatric neuropsychologist, she specializes in rare neurodegenerative disorders of childhood, evaluating functional course of disease and response to therapy. Her research has focused on the MPS disorders, particularly examining outcomes of early diagnosis, newborn screening, and novel therapies. Her research extends to defining and measuring aspects of disease that are under-represented in the clinical and research communities but are important and meaningful to patients and families, such as neurobehavioral symptoms and the family lived experience. Her recent work has examined measuring the effects of new therapeutic approaches on these priority symptoms. Dr. Eisengart has been a principal or co-investigator, and research mentor, on a number of observational studies or clinical trials for MPS and other rare diseases. She has served on the Technical Expert Panel for the U.S. Secretary of Health and Human Services to consider the addition of MPS II to the Recommended Uniform Screening Panel (RUSP), and on the Working Group for the Minnesota Department of Health to implement newborn screening for MPS I and other rare disorders. Presently she sits on the National Organization for Rare Disorders (NORD) Training Work Group and the Critical Path Institute Rare Disease Clinical Outcome Assessment Consortium.

	Matthew Ellinwood DVM, PhD, CSO National MPS Society Dr. Matthew Ellinwood is the Chief Scientific Officer at the National MPS Society, where he guides research to benefit the patient community. He is a comparative medical geneticist, with over two decades of research experience with the mucopolysaccharidosis and mucolipidosis type II/III disorders. He has a long association with the Society beginning as a Scientific Advisory Board member in 2005. He is a Professor Emeritus (Iowa State University), where he conducted basic and applied research on the neuropathic MPSs. He is experienced in preclinical hematopoietic stem cell transplantation, intravenous enzyme therapy, intracisternal enzyme therapy, intraventricular enzyme therapy, systemic intravenous AAV based gene therapy, and intraparenchymal CNS directed AAV based therapy. In addition to administration of the Research Grants Program, he promotes multiple initiatives, from public health base newborn screening to drug evaluation and approval policy.

	Theresa Ferlita CareConnect PSS Patient Education Liaison, Rare Diseases at Sanofi Theresa (Terry) Ferlita is a PEL with Sanofi and currently covers the North Florida area. She has been a PEL for 12 years and provides education and support to patients and families living with a rare disease. Terry is a certified and licensed genetic counselor and lives in Tampa, Florida. She is married, has one son, and enjoys some traveling and the beaches.

	Amy Gaviglio MS, CGC Amy Gaviglio is a genetic counselor and founder of Connetics Consulting, LLC which provides newborn screening, genomics, and rare disease services globally. She has worked in the NBS and rare disease space for the past 17 years. Amy currently works with the Centers for Disease Control and Prevention, the Association of Public Health Laboratories (APHL), Expecting Health, RTI International, and several other rare disease and genomics organizations. She is co-chair of APHL’s New Disorders in Newborn Screening Subcommittee and is a member of additional national groups including the Rare Disease Diversity Coalition and EveryLife Foundation’s Community Congress. She also serves on the Executive Board for the International Society of Neonatal Screening and is a member of the MPS Society’s Scientific Advisory Board. Finally, Amy serves as Chair of the NBS Expert Panel for the Clinical and Laboratory Standards Institute.

	Michael Gelb PhD

	Ozlem Goker-Alpan PhD Dr. Ozlem Goker-Alpan is the founder and president of LDRTC, a non-profit organization focusing on Lysosomal Disorders and other rare diseases. She established LDRTC in 2013 with the vision of providing quality care to individuals with rare diseases by offering clinical care and translational research under one roof. Dr. Goker-Alpan obtained her medical degree in 1990 from Marmara University School of Medicine in Istanbul, Turkey, graduating top of her class. She completed her Pediatrics training as a Pediatric Chief Resident at SUNY Stony Brook, New York. She then pursued her first Clinical and Biochemical Genetics fellowship at the National Institutes of Health, Greater Washington Medical Genetics Program, in 1999. She served as an adjunct scientist at the National Child Health Institute. Following this, her second fellowship focused on Lysosomal Storage Disorders and Gaucher disease at the Clinical Neuroscience Branch, National Institute of Mental Health (NIMH), NIH. She also coordinated the NIH Gaucher Clinic at the Medical Genetics Branch of the National Human Genome Research Institute (NHGRI). Dr. Goker-Alpan's commitment to the future of healthcare in the field of Lysosomal Storage Disorders is evident in her dedication to education and training. As an established clinician and translational scientist, she is focused on providing individualized care and treatment for patients with LSDs and rare diseases. Under her leadership, LDRTC has completed multiple scientific projects exploring immune pathways and lysosomal functions to develop new diagnostic and monitoring tools in LSDs and GBA-related Parkinsonism. She also serves on the scientific advisory boards of multiple pharmaceutical companies and patient advocacy organizations.

	Jennifer Goldstein PhD, CGC Jennifer (Jenny) Goldstein received her PhD in medical genetics from the University of Aberdeen, Scotland. She then moved to the University of California - Berkeley, to carry out post-doctoral studies in Molecular and Cell Biology and went on to complete a master’s program in Genetic Counseling at Virginia Commonwealth University. Jenny worked as a pediatric genetic counselor at Duke University Medical Center, seeing patients and conducting research on glycogen storage diseases and creatine deficiency syndromes. Now, as an assistant professor of Genetics at the University of North Carolina at Chapel Hill, Jenny works with the Clinical Genome Resource (ClinGen), a NIH-funded effort to create a publicly available resource on gene-disease clinical validity and the pathogenicity of genetic variants. Jenny is a member of various ClinGen gene and variant curation expert panels, focusing on inborn errors of metabolism and ocular disorders.

	Frannie Hoffman Frannie Hoffman is a counselor, spiritual intuitive, writer, and artist, and an extraordinary facilitator. Through her meditation circles, writing, and compelling lectures and seminars, she motivates and empowers people all over the USA and Canada by her personal and honest expression of life and Divine Spirit. She has devoted her life to helping others tap into their own spiritual power, to take responsibility, to create and inspire personal empowerment, and to experience themselves as Divine beings. Nothing prepared Frannie for the sudden loss of her husband when he dropped on the floor from an aneurism. None of her personal or professional experiences prepared her for what lay physically, emotionally, or spiritually in the wake of Steve’s transition. Writing this book, Loss: The Doorway to New Life was an integral part of her grieving process. Frannie has also written a memoir, From Modeling Clothes to Modeling Self. A truly empowering read as her compelling story opens your heart to love and ultimately bring you to who you are as a divine being. Visit my other website www.franniehoffman.com

	Steve Holland MPS I Parent, Vice Chairman, National MPS Society Steve Holland, CPA, is the proud father of Spencer, Madison and Laynie, who were diagnosed with attenuated MPS I in 1994. The Hollands live in Texas, where they host a successful MPS fundraiser annually. Steve has served on the Society’s board since 1998, formerly serving as Chairman and Treasurer and currently serving as Vice-Chairman. He chairs the Education/ Publicity Committee and the Pathways Committee and is a frequent speaker on MPS and rare disease topics.

	Carl Kapes National MPS Society Carl is dad to Ryan (forever 16, MPS IIIA), Brayden (MPS IIIA), Bryce, and Brooklyn. He lives in Wilmington, DE. Carl works as an project/electrical engineering manager for Burns Engineering in Philadelphia, PA. He attended Villanova University where he majored in Electrical Engineering and played on the Wildcats baseball team. He has raised more than $1 million for Sanfilippo syndrome through various fundraisers. Carl summitted Mt. Kilimanjaro in 2012, Mt. Rainier in 2016, and Pico de Orizaba in Mexico in 2018, all as fundraisers. In addition to the National MPS Society, Carl is also a board member of the Team Sanfilippo Foundation and Naamans Little League in Wilmington, DE

	Terri Klein President and CEO Terri Klein, is the President and CEO at the National MPS Society and has spent two decades in rare disease advocacy. She was the first Executive Director for ISMRD and for the last 16 years in Development, Operations, and now CEO at the National MPS Society. Her expertise is in organizational development, multi-tier fund development, and patient advocacy. She is a Board Director of the International MPS Network, and a member of CPAG, part of the LDN. IMPSN focuses on global therapeutic access and humanitarian outreach. Terri is a founding member of the Mucolipidosis Research Collaborative Network. Her graduate degrees are in Public Administration and Nonprofit Management. Terri’s advocacy efforts have been published. In addition to oversight of the Society, her initiatives include health equity issues, drug development, and numerous rare disease policy issues. She is the mother of Jennifer, ML III and has been a member of the Society since 2001.

	Heather Lau MD, MS, Executive Director of Global Clinical Development at Ultragenyx Heather Lau is the executive director of Global Clinical Development at Ultragenyx, a company focused on development of therapies for rare genetic diseases. Dr Lau joined industry in 2021 after 10 years in academia where she was the Director of the Lysosomal Storage Disorders Program and the Associate director of Neurogenetics at NYU School of Medicine. She studied biology as an undergraduate at Cornell University, and holds a masters in biochemistry and molecular biology from New York Medical College. She obtained her medical degree from University of Rochester. She later trained in pediatrics at Montefiore Medical Center; and trained in neurology and neurogenetics at NYU School of Medicine. During her time at NYU, she ran an undiagnosed disease clinic to aid in the diagnosis and management of suspected inherited neurodegenerative diseases for both adults and pediatric patients. Her clinical research included investigating a range of modalities including small molecule (chaperones and substrate reduction inhibitors), protein (enzyme)-based and both systemic and CNS directed gene therapy for a variety of lysosomal disorders. She continues her academic career as adjunct assistant professor at Yale University in the Department of Internal Medicine.

	Angela LeDay PharmD, PhD, Sr. Director Medical Science Liaison at Denali Therapeutics Angela LeDay is a Senior Director in Medical Affairs at Denali Therapeutics. Angela has over 20 years of medical affairs experience and has worked across a variety of therapeutic areas in pharma and biotech industry. At Denali, Angela leads a field-based medical team focused on providing scientific information and medical support for lysosomal storage disorders. Angela has a Doctor of Pharmacy degree and PhD in neuropharmacology.

	Robin LeWinter PhD, USA Medical Affairs & Medical Communications Lead at JCR USA, Inc Robin LeWinter has dedicated her professional life to advancing therapeutic strategies for underserved patient populations, focusing particularly on rare disease neurometabolic disorders. Throughout her career, Robin has played a pivotal role in collaborating with researchers, healthcare professionals, and regulatory bodies to enhance product development and ensure patient access to innovative therapies. She remains committed to fostering collaboration and advancing knowledge within the rare disease community, making a meaningful impact on the lives of patients and their families.

	Nathalie Marie MD Dr. Nathalie Marie was born and raised in Miami, FL and currently resides outside of Boston, MA. She completed her BA degree at Boston College in Massachusetts where she double majored in philosophy and pre-medical sciences. There, she graduated Magna Cum Laude and was the recipient of the Dean’s Scholar Award. She attended medical school at the Miller School of Medicine in Miami, FL and was the recipient of the Distinction in Psychiatry Award selected by faculty as having both academic and clinical distinction within a graduating class. Dr. Marie completed her psychiatric residency in 2008 in Houston, TX at Baylor College of Medicine Menninger Department of Psychiatry. She received multiple distinctions during her residency training including the Association of Women Psychiatrist Alexandra-Simmons Fellowship Award for distinction in women’s mental health and the Dr. Henry Page Durkee Laughlin Foundation Merit Award. After residency, Dr. Marie served the veteran population for 6 years at the Michael E. DeBakey VA Medical Center where she also held dual appointment with Baylor College of Medicine. There she worked as an outpatient psychiatrist within the Trauma and Recovery Program specializing in post traumatic stress and anxiety disorders, as well as other mood disorders. In 2011, she was a Federal Executive Horizon Award Nominee in the category of leadership. Dr. Marie has since worked in community psychiatry and in private practice and currently works as a telehealth outpatient psychiatrist. She holds special interests in trauma, women's mental health and in working with adults with differing abilities and their families . She is fluent in three languages, enjoys ballroom dancing, travel and spending time with her husband and 12 year old son who was diagnosed with MPS II at age 3.

	Joseph Muenzer MD, PhD, University of North Carolina Medical Genetics Joseph Muenzer, MD, PhD, is the Bryson Distinguished Professor in Pediatric Genetics and a Professor in the Department of Pediatrics and Department of Genetics at the University of North Carolina at Chapel Hill, where he has practiced since 1993. He received an MD (1976) and PhD in biochemistry (1979) from Case Western Reserve University in Cleveland, OH. He completed a residency in pediatrics at the University of Wisconsin Hospitals, Madison, and a genetic/endocrine fellowship at the National Institute of Child Health and Human Development, NIH, in Bethesda, MD. Dr. Muenzer is the Director for the recently created Joseph Muenzer MPS Research and Treatment Center at the University of North Carolina at Chapel Hill. An academic based Mucopolysaccharidoses (MPS) Center created to improve the lives of MPS individuals and their families. Dr. Muenzer is involved in the diagnosis, management, and treatment of patients with inborn errors of metabolism, especially the mucopolysaccharidoses and newborn screening for MPS I and MPS II. He is board certified in Pediatrics and in Clinical Biochemical/Molecular Genetics. He has been actively involved in developing new treatments for MPS disorders his entire professional career. He has created a mouse model for Hunter syndrome (MPS II) that has been widely used to develop new treatment for MPS II. He has been a principal investigator (PI) for IV enzyme replacement clinical trials (ERT) for both MPS I and MPS II resulting in FDA approval. His recent clinical research has focused on the development of new treatments for the brain disease in MPS. Dr. Muenzer was the PI for Phase I/II and Phase II/III intrathecal enzyme replacement clinical trials for severe MPS II and now the post-trial access PI. He has been the principal investigator for >20 MPS clinical trials/observational studies. Dr. Muenzer is currently the PI for a Phase I/II gene editing clinical trial for MPS II and a Phase I/II and phase II/III IV ERT clinical trials to treat the brain disease in MPS II.

	Dawn Phillips PT, MS, PhD, Senior Director of Clinical Outcomes Research at REGENXBIO Dawn Phillips PT, MS, PhD is the Senior Director of Clinical Outcomes Research at REGENXBIO Inc. Dr Phillips received her doctorate in Human Movement Science from UNC Chapel Hill with a focus on motor learning and motor control and principles of outcome measurement. She was a fellow in the Leadership Education in Neurodevelopmental Disabilities (LEND) program and completed a certificate in Core Public Health Concepts. Dr Phillips also has a MSc in Human Movement Science with a focus on Infant and Family Studies and a BSc in Physical Therapy. She has been working in in rare disease clinical research for 23 years with a focus on research design and endpoint development, qualitative research, concept elicitation and validation, site training, data analysis and regulatory submission and engagement. She also was an assistant professor at UNC Chapel Hill with a course instruction focus on evidence-based medicine and pediatric outcomes. Dr Phillips’s work has appeared in multiple journals including Molecular Genetics and Metabolism, Neuromuscular Disorders, Value in Health, Human Gene Therapy, Neurology, and the Journal of Pediatric Rehabilitation Medicine.

	Roberto Pineda MD Dr. Roberto Pineda completed his undergraduate and medical degree at the University of Minnesota Medical School, where he was elected to AOA. He was selected for the Howard Hughes NIH-HHMI Research Scholars Program at the Cloisters in Bethesda Maryland where he worked with retinoblastoma cell lines at the National Eye Institute. He went on to complete his residency in Ophthalmology at the Massachusetts Eye and Ear Infirmary, Harvard Medical School where he served as Chief Resident and Director of the Trauma Service. As a Heed Fellow recipient, he completed his Cornea and Refractive Surgery Fellowship at the Massachusetts Eye and Ear Infirmary. Currently, he is The Thomas Y and Clara W Butler Chair in Ophthalmology and an Associate Professor of Ophthalmology at Harvard Medical School, serving as full-time faculty of the Cornea and Refractive Surgery Service at the Massachusetts Eye and Ear Infirmary. Additionally, he was the former Division Chief of Ophthalmology at the Brigham and Women’s Hospital and Director of Refractive Surgery. Dr. Pineda regularly lectures nationally and internationally at our professional meetings, including several named lectures and has published over 100 peer-reviewed papers and co-authored 5 books including the award-winning Massachusetts Eye and Ear Infirmary Illustrated Manual of Ophthalmology (5th ed.) and the Essentials of Cataract Surgery (2nd ed). He has received both the American Academy of Ophthalmology (AAO) Achievement and Senior Achievement Awards and has served on the AAO Practicing Ophthalmology Committee in Cataract, AAO Ethics Committee and the AAO Preferred Practice Pattern, Adult Cataract. In 2017, he was elected and induced to the International Intraocular Implant Club (IIIC), an international ophthalmological society. He currently sits on the Board of KeraLink International working to reduce corneal blindness through advancements in novel low resource technologies. More Recently, in 2021, he was selected Surgical Teacher of the Year by the ophthalmology residents at the Massachusetts Eye and Ear Infirmary and at the 2023 AAO meeting, he was the recipient of the ISRS Jose Barraquer Lecture and Award, honoring a physician who has made significant contributions in the field of refractive surgery.

	Lynda Polgreen MD Dr. Lynda Polgreen is a pediatric endocrinologist who earned her medical degree from the Mount Sinai School of Medicine. Following her degree, she completed a pediatric residency and a postdoctoral fellowship in pediatric endocrinology at the University of Minnesota. Currently, Dr. Polgreen serves as an Investigator at The Lundquist Institute at Harbor-UCLA Medical Center and holds the role of Associate Professor of Pediatrics at the David Geffen School of Medicine – UCLA. She practices as a Pediatric Endocrinologist at Harbor-UCLA and Children’s Hospital Orange County, where she contributes to a multidisciplinary MPS clinic. In addition, she is the Clinical Director of the Center for Treatment of Rare Diseases and the Associate Director of the Translational Genomic and Population Science Institute at The Lundquist Institute. Dr. Polgreen's research is focused on advancing the treatment and understanding of the mucopolysaccharidoses, with a particular emphasis on exploring immune dysfunction within this group of disorders. She leads clinical drug repurposing trials that target the downstream effects of lysosomal dysfunction, aiming to improve bone, joint, and central nervous system outcomes for patients with MPS.

	Manas Prasad PharmD, Medical Science Liaison at Orchard Therapeutics Manas Prasad, PharmD, is the Medical Science Liaison (MSL) for the Eastern US at Orchard Therapeutics. He started at Orchard in 2019 as the lead for their Medical Information department and oversaw the Compassionate Use process. He later transitioned to the field-based MSL role where he partners with health care providers to exchange scientific information, better understand Orchard’s disease state interests, and educate external audiences about our commercial and pipeline gene therapies.

	Curran Simpson President and Chief Executive Officer, Board Member atREGENXBIO Curran M. Simpson is the President and Chief Executive Officer and member of the Board of Directors at REGENXBIO. Mr. Simpson previously served as the Company’s Chief Operating Officer. In that role, he led key business functions including Research & Clinical Development, Corporate Strategy, Manufacturing & Quality, Regulatory, and Commercial Operations. Mr. Simpson joined REGENXBIO in 2015 with extensive leadership experience across biopharmaceutical operations and served as the Company’s Chief Technology and Operations Officer before becoming COO. Prior to joining REGENXBIO, he was the Regional Supply Chain Head for North America and Interim Chief Operating Officer at GlaxoSmithKline (GSK). Mr. Simpson earlier served as interim CEO of Human Genome Sciences (HGS), where he led the integration of HGS into GSK, and as Senior Vice President of Operations and Vice President of Manufacturing Operations at HGS. Prior to HGS, Mr. Simpson was Director of Manufacturing Sciences at Biogen. Earlier in his career, Mr. Simpson served in an overseas assignment at Novo-Nordisk Biochem in Denmark and in various senior development and engineer roles at Genentech, working on Herceptin and Avastin, among other roles. Mr. Simpson has an M.S. in surface and colloid science from Clarkson University and a B.S. in chemistry from the Clarkson College of Technology.

	Lisa Todd MPS II Parent, Chairman of the Board, National MPS Society Lisa Todd lives in Albuquerque, NM, with her husband, Jerry, and their three sons—Jack (MPS II), Jake, and Jaden. Lisa supports the Society by serving on the board, hosting the Keep Hope Alive fundraiser, and serving on the Fundraising and Governance committees. Lisa is a CPA and specializes in not-for-profit entities. Through her professional role, she provides training to many types of non-profits on a variety of governance and financial issues. Lisa has been a member of the Society since Jack was first diagnosed with MPS in June 2011 and has been a board member since January 2012.

	Douglas von Allmen MD Douglas C. von Allmen, MD Assistant Professor Department of Otolaryngology – Head and Neck Surgery University of Cincinnati Academic Health Center Cincinnati Children’s Hospital Medical Center Medical School: Joan C. Edwards School of Medicine, Marshall University, Huntington, WV 2010-2014 Residency: University of Cincinnati College of Medicine, Cincinnati, OH, Otolaryngology Head and Neck Surgery, 2014-2019 Fellowship: Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, Pediatric Otolaryngology, 2019-2021 Board Certification: American Board of Otolaryngology Clinical Interests: General Pediatric Otolaryngology, Esophageal reconstruction, Tracheoesophageal fistula, Tracheomalacia Dr. von Allmen is an Assistant Professor in the Division of Pediatric Otolaryngology at Cincinnati Children’s Hospital Medical Center and the University of Cincinnati Academic Health Center. He joined the faculty in 2021 after completing residency and fellowship in Cincinnati. Dr. von Allmen participates in the aerodigestive multidisciplinary center as well as the complex obstructive sleep apnea center. His interests include airway and esophageal reconstruction, tracheomalacia, and tracheoesophageal fistula.

	Ryan Watts PhD, Denali Therapeutics Ryan Watts is the Chief Executive Officer of Denali Therapeutics. He is a co-founder, President and a member of the Board of Directors. Under Watts’ leadership, Denali has advanced multiple therapeutic candidates into clinical studies for Parkinson’s disease, Hunter syndrome, Sanfilippo syndrome, and ALS. Denali has invented a proprietary blood-brain barrier platform for delivery of therapeutic proteins to the brain. Watts has also led efforts to raise significant capital to advance Denali’s therapeutic pipeline and has been instrumental in forging partnerships to accelerate the discovery and development of medicines for lysosomal storage disorders and neurodegeneration. Watts previously served as Director of the Department of Neuroscience at Genentech. During his tenure there, he led the company’s re-entry into neuroscience. The Watts laboratory focused on drug discovery for cancer and Alzheimer’s disease, with an emphasis on understanding mechanisms of neurodegeneration guided by human genetics. His lab also studied various aspects of blood-brain barrier biology and delivery. Watts obtained his Ph.D. from Stanford University’s Department of Biological Sciences and his B.S. in Biology from the University of Utah. Watts lives in Salt Lake City, Utah with his family. Connecting with individuals and families impacted by the diseases Denali focuses on is incredibly meaningful and fuels his purpose for the work we do at Denali.

	Klane White MD, MSc, Colorado Children's Hospital Klane K. White, MD, MSc is Chair of the Department of Pediatric Orthopedics and the Rose Brown Endowed Chair of Pediatric Orthopedic Surgery at Children’s Hospital Colorado. He is Professor of Orthopedics and Vice Chair in the Department of Orthopedics at the University of Colorado School of Medicine, where he is also Professor of Genetics and Metabolism in the Department of Pediatrics. Dr. White is a graduate of the University of Southern California where he earned his bachelor and master’s degrees in Biology and Biological Oceanography, respectively. He received his Doctor of Medicine from the George Washington University in Washington, DC. Dr. White completed his surgical internship and orthopedic surgery residency at the University of California San Diego, as well as a two-year NIH sponsored fellowship in orthopedic research. He received advanced fellowship training in pediatric orthopedics and scoliosis at Texas Scottish Rite Hospital for Children. A renowned leader in his field, Dr. White is an internationally recognized expert and advocate in the care of mucopolysaccharidosis and skeletal dysplasia. He serves on the Medical Advisory Board of Little People of America, the Scientific Advisory Board of the National MPS Society, and is a principal investigator for multiple international research studies on emerging treatments in rare disease. Dr. White is also a Principal Researcher and Research Interest Group leader in the Pediatric Spine Study Group, and an executive founding member and current president of the Skeletal Dysplasia Management Consortium. In addition to skeletal dysplasia and the mucopolysaccharidoses, Dr. White’s clinical and research interests also include the diagnosis and management of early onset scoliosis, metabolic bone disease, and complex spine deformity. Dr. White has authored more than 100 peer-reviewed publications, review articles and book chapters, and serves as a standing reviewer for both orthopedic and rare disease journals.

	Tom Wier Social Security Administration Married for 37 years to Blanca Wier, proud father of four sons, including Frankie (MPS II) who passed away in 2014. Retired dual service (Army/Navy) veteran of 22 years whose final tour included serving with a counterintelligence unit at Fort Sheridan, IL/Fort Meade, MD. After receiving an Honorable Discharge from the U.S. Army in 2009, has been presently employed as a Representative for the Social Security Administration in Woodstock, IL, serving the community, performing in depth analysis of benefits and answering any specific questions and concerns from the public at large.