Speakers

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.

JP Clancy, MD
Senior Vice President, Clinical Research for the Cystic Fibrosis Foundation (CFF)
JP Clancy is a pediatric pulmonologist and the Senior Vice President of Clinical Research for the Cystic Fibrosis Foundation (CFF). Previously he served as the division director of Pediatric Pulmonology at the University of Alabama at Birmingham (2003-2010) and the director of research for the Pulmonary Medicine Division at Cincinnati Children’s Hospital Medical Center (2011-2018). His CFF roles include oversight of multi-center clinical research conducted by the CFF Therapeutic Development Network, CFF support of global clinical research networks, investigator-initiated clinical research programs, physician-scientist training and career development and clinical research resources.
Clancy has published over 200 peer reviewed manuscripts with a focus on clinical research outcome measures and airway epithelial biology.

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.

Nathan Grant, MPhil
Founder, Siblings with a Mission
President, Siblings with a Mission
Nik's brother
Nathan Grant is the Founder and President of Siblings with a Mission, an international organization that supports siblings of people with complex health conditions and their families. He has also served on the Board of Directors of Project Alive and is the author of several research articles on rare diseases, including the book The MPS Sibling: Short Stories for Brothers and Sisters.
His inspiration comes from his twin brother Nik, who has a rare, genetic condition called MPS II (also known as Hunter syndrome). From Cincinnati, Ohio, Nathan is currently a third-year MD/MBA student at Harvard Medical School and Harvard Business School. He received his undergraduate degree in biology from Harvard College and an MPhil in Health, Medicine and Society from the University of Cambridge as a Harvard-UK Fellow. Inspired by his brother, Nathan hopes to help improve outcomes for people with rare conditions and their families through clinical care, research and advocacy.

Eric Green, MD, PhD
Director, National Human Genome Research Institute (NHGRI)
Eric Green is the director of the National Human Genome Research Institute (NHGRI) at the U.S. National Institutes of Health (NIH). He is the third NHGRI director, having been appointed by NIH director Francis Collins, MD, PhD in 2009.
Green has been at the Institute for nearly 30 years, during which he has had multiple key leadership roles. He served as the institute’s scientific director for 7 years, chief of the NHGRI Genome Technology Branch for 13 years and founding director of the NIH Intramural Sequencing Center for 12 years.
For just over two decades, Green directed an independent research program that included integral start-to-finish roles in the Human Genome Project and groundbreaking work on mapping, sequencing and characterizing mammalian genomes.
Green earned his MD and PhD degrees in 1987 from Washington University in St. Louis; coincidentally, the word “genomics” was coined in that same year. Throughout his career, he has authored and co-authored over 390 scientific publications. Green has earned several honors and awards, including election to the National Academy of Medicine in 2023.

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
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.

Genny Jessee
Executive Director, June Jessee Memorial Foundation
Co-founder, June Jessee Memorial Foundation
June's mom
Genny Jessee is the executive director of the June Jessee Memorial Foundation (JJMF), a nonprofit organization dedicated to providing relief and support to children with medically complex, neurological conditions and their families, offering resources, services and connections to help make their lives easier. She and her husband, Matt Jessee, founded the JJMF in 2016 in honor of their brave daughter, June Jessee, who fearlessly faced the multiple, life-limiting conditions she endured.
Genny leads all of the JJMF’s fundraising and programming to support children with medically complex, neurological conditions and their families. She curates and shares important resources and information through the JJMF website which has become a leading online resource to help parents and caregivers in the St. Louis area and beyond to navigate these chronic, neurological conditions with no specific diagnosis amid a complicated healthcare landscape.
As the lead editor and writer for the JJMF’s blog, Sorry to meet you this way, Genny’s candid and heartfelt blog posts bring to life the challenges, concerns, joys, and emotions families of children with undiagnosed, neurological conditions face, tackling a wide-variety of topics. Genny’s blog is frequently featured on TODAY Parents and Scary Mommy, as well as international parenting websites. Additionally, she has created both online and in-person opportunities for parents to meet and make connections with one another, finding comfort and support from those who understand the significant obstacles of the conditions.
In 2020, Genny was honored with the Missouri Athletic Club Woman of Distinction Award in recognition of the positive impact she has made in the region. She additionally was a finalist for the “Best Kept Secret” in the WEGO health awards for her contribution of impactful content in the online health community.
Previously, Genny held public relations and communication positions for leading agencies in Washington, DC She is the mom of two young boys and enjoys spending time with her family, journaling, exercising, needlepointing, reading and binge watching her favorite TV shows.

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
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
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
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.