The Professional Advisory Council acts as a liaison between the patient advocacy triangle: industry, providers, and the patient community, where we share information and communicate openly to find the most urgent unmet needs of patients and find ways to fill those needs. The Council is available to provide assistance and meets on an as-need basis.
Associate Professor, Division of Clinical and Translational Genetics, Dr John T. Macdonald Foundation Department of Human Genetics, University of Miami, Miller School of Medicine
Faculty, New York University School of Medicine
Diplomate American Board of Pediatrics
Stuart H. Ditchek, MD, is a board-certified pediatrician, educator, author, and physician leader whose career has focused on pediatric medicine, Jewish genetic diseases, rare disorders, and the care of medically complex children and young adults. Trained at SUNY Downstate/Kings County Hospital Center, where he served as chief resident, Dr. Ditchek has held academic appointments at NYU Langone Medical Center and Maimonides Medical Center and serves on faculty as a Clinical Assistant Professor of Pediatrics at NYU School of Medicine. He previously served as Associate Director of NYU’s Division of Familial Dysautonomia under its founder, Dr. Felicia Axelrod, and in 2018 received the NYU School of Medicine 25-Year Faculty Service Achievement Award. His extensive work in Jewish genetic diseases has included service as Medical Director of the Jewish Genetic Disease Consortium, development of DNA-based community screening initiatives, and the establishment of JPatible, a genetic screening and matching program. His subsequent work in rare diseases has included participation in the Sanofi/Genzyme Rare Disease Speaker’s Bureau and participates in their United States Gaucher Advisory Board. His clinical practice includes the care of children and adults with Gaucher disease and other genetic disorders, and in 2026 he joined the professional advisory board of the GCA professional advisory council.
Dr. Ditchek has also dedicated much of his career to improving care and quality of life for children with serious illness and disabilities. After serving as founding Medical Director of Chai Lifeline’s Camp Simcha Special, he co-founded Kids of Courage in 2008, a nonprofit providing medically supervised travel and year-round programming for seriously ill children at no cost to their families. His expertise in caring for and transporting medically fragile individuals has included advisory work with the Transportation Security Administration. During 9/11 attacks, he spent several days at the triage area of the south tower Trade Center collapse providing emergency care to first responders and victims. During a 2002–2003 sabbatical at Shaare Zedek Medical Center in Jerusalem, he joined Magen David Adom’s First Responders Unit and participated in mass-casualty and emergency response. This included specialized training in nuclear, biological and chemical mass casualty response during the 2003 Iraq war. Since 2016, he has served as an honorary police surgeon with the New York City Police Department. Dr. Ditchek maintains a private pediatric practice in Brooklyn, New York.
Fairfax, Virginia
Genetic Counselor IV/Study Coordinator
UPMC Children’s Hospital of Pittsburgh
University of Pittsburgh
Nadene Henderson is a genetic counselor who began her lifelong dedication to Lysosomal Storage Disorders (LSD) at the University of Pittsburgh in September of 1994. Her involvement over the years with many different research studies and clinical care, has provided the opportunity to see patients at Children’s Hospital of Pittsburgh, Montefiore, Presbyterian and Magee-Women’s Hospitals, all under the original mentorship of Dr. John Barranger and Erin O’Rourke, MS, CGC. She is dedicated to providing access to all families impacted by lysosomal storage disorders with quality, individualized care on both the clinical and research side. Her current main focus is study coordination, clinical trials for all lysosomal storage disorders, newborn screening, and local and national education in the field of lysosomal storage diseases, alongside the other members of our LSD team, and under the direction of Damara Ortiz, MD. Outside of work, Nadene enjoys travelling to new locations and continually improving to be the best mom and wife she can be to her three children and husband.
Lysosomal Storage Disorders Program | UPMC Children’s Hospital
Assistant Professor, University of Minnesota, Center for Orphan Drug Research, Dept. of Experimental and Clinical Pharmacology
Professor Emeritus Division of Medical Genetics Department of Pediatrics, University of California, San Francisco
Chief, Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health
