Philip (PJ) J. Brooks, PhD

Philip (PJ) J. Brooks, PhD

Acting Director
Division of Rare Diseases Research Innovation
National Center for Advancing Translational Sciences
National Institutes of Health (NIH)

Philip J. (PJ) Brooks is the acting director of NCATS’ Division of Rare Diseases Research Innovation. Brooks represents NCATS in the NIH-wide Gene Therapy Working Group, the Regenerative Medicine Innovation Project and the International Rare Diseases Research Consortium (IRDiRC). He also is the working group co-coordinator for the NIH Common Fund program on Somatic Cell Genome Editing, one of the leaders of the Platform Vector Gene Therapy (PaVe-GT) pilot project and the co-chair of the Bespoke Gene Therapy Consortium.

Kacie Craig, MEd

Kacie Craig, MEd

Executive Director
The Cute Syndrome Foundation

Kacie Craig, MEd is the Executive Director of The Cute Syndrome Foundation. Her daughter, Stella, was diagnosed with an ultra-rare genetic disorder called SCN8A at five months old and it was then her passion for advocacy was ignited. Craig began volunteering for TCSF in 2018 and joined the foundation's executive leadership team in 2020. It was in early 2022 that she transitioned from her long standing career in radiography education to a full time advocate. Kacie and Stella have both been featured in epilepsy awareness segments on KMOV Channel 4 News in St. Louis, Missouri and KY3 News in Springfield, Missouri.

Sara Hamilton

Sara Hamilton

Clinic Manager
Department of Neurology, Washington University School of Medicine

Sara Hamilton is the Clinic Manager for the Department of Neurology at Washington University School of Medicine, where she oversees clinical operations for the Department’s outpatient pediatric clinics. Sara is also the mother of a child with Fragile X Syndrome who has worked closely with local, state, and national organizations focused on Fragile X education and advocacy for more than a decade. Sara has served as the Vice President and President of the Fragile X Resource Center of Missouri, a Community Support Network Leader with The National Fragile X Foundation, and as a Family Ambassador for FRAXA, the national organization focused on advancing Fragile X research.

Michele Herndon, MSN, RN, CPEN, TCRN, TNS

Michele Herndon, MSN, RN, CPEN, TCRN, TNS

Vice-President of The Mitchell and Friends Foundation

Michele Herndon is the co-founder and vice-president of the Mitchell and Friends Foundation.  The foundation raises awareness of Mitchell Syndrome, promotes research into possible treatments, and offers support to patients and families affected by Mitchell Syndrome.  This newly discovered disease was discovered in part through information learned from Michele’s oldest son, Mitchell, who was the first patient identified.  Professionally, Michele has been a pediatric nurse since 2005 and is currently a nursing manager for St. Louis Children’s Hospital in pediatric trauma and injury prevention.

Peter Marks, MD, PhD

Peter Marks, MD, PhD

Director
Center for Biologics Evaluation and Research (CBER)
U.S. Food and Drug Administration (FDA)

Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry on drug development and is an author or co-author of over 100 publications. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in 2016. Over the past several years he has been integrally involved in the response to various public health emergencies and in 2022 he was elected a member of the National Academy of Medicine.

Edward Neilan, MD, PhD

Edward Neilan, MD, PhD

Chief Medical and Scientific Officer
National Organization for Rare Disorders (NORD)

Dr. Edward Neilan is the Chief Medical and Scientific Officer at the National Organization for Rare Disorders (NORD®). Ed is a molecular biologist, pediatrician, and medical and biochemical geneticist. He has also worked on many rare disease clinical trials and has contributed to the clinical development and FDA-approval of several new rare disease therapies. Immediately prior to joining NORD, Ed worked at Sanofi Genzyme, where he led global medical affairs for rare neurogenetic diseases and also contributed to clinical development efforts across multiple programs. Prior to that, Dr. Neilan served a term as President of the Medical Staff at Boston Children’s Hospital. In addition to his full-time role at NORD, Dr. Neilan also still maintains part-time faculty appointments at Boston Children’s Hospital and Harvard Medical School.

Holm Schneider, MD

Holm Schneider, MD

Head, Center for Ectodermal Dysplasias
Department of Molecular Pediatrics
University Hospital Erlangen, Erlangen, Germany

Holm Schneider obtained his MD from the University of Leipzig and received German board certifications in general pediatrics, emergency medicine and neonatology. He was research fellow at Imperial College London, U.K., registrar at the University Hospital Erlangen, Germany, and Professor of Experimental Neonatology at Innsbruck Medical University, Austria, before returning to the University Hospital Erlangen where he has been working as Professor of Pediatrics since 2008. He is head of the interdisciplinary Center for Ectodermal Dysplasias Erlangen, the German national reference center for ectodermal dysplasias and p63-associated disorders. The research of his team is dedicated to novel therapeutic approaches to severe genodermatoses. Schneider pioneered the prenatal drug therapy of genetic diseases and conducted numerous preclinical and investigator-initiated or industry-funded clinical studies on life-threatening congenital disorders. He received prestigious honors and awards, including the Gottron-Just Science Prize, the Gold medal of the Union of European Neonatal and Perinatal Societies, and the Care-for-Rare Science Award.

Tracy VanHoutan

Tracy VanHoutan

President: Noah’s Hope – Hope4Bridget Foundation
Former Board Member and VP (6 years): Batten Disease Support & Research Association (BDSRA)

Tracy is involved with advocating for all rare diseases within various agencies and with members of Congress. Tracy has spoken before the FDA on multiple occasions, been invited as a panelist for cell based therapies at the NHLBI within the NIH, and has been a featured panelist at a Rare Disease Congressional Caucus hearing on Capitol Hill.  Over the years Tracy & Jennifer have worked with many other patient advocates to advance legislation including the 2012 FDASIA bill and the 2016 21st Century Cures Act.