A Day in the Life: Inside the Research Participant Experience at Weill Cornell Medicine скачать в хорошем качестве

A Day in the Life: Inside the Research Participant Experience at Weill Cornell Medicine

Thinking about joining a clinical trial for Ataxia but unsure what it really involves? The National Ataxia Foundation is proud to host this webinar presented by Madeline Galbraith, BA from the The Weill Cornell Medicine Department of Genetic Medicine on May 29th, 2025. In "A Day in the Life: Inside the Research Participant Experience at Weill Cornell Medicine," the lab's team will take you behind the scenes and give you an inside look at the research participant experience, with a focus on their Friedreich’s Ataxia (FA) gene therapy trial. Their expert coordinator will walk you through the essentials -- study visits, travel support, costs (spoiler: none!), and what to expect at every step. You’ll also meet Dr. Ronald Crystal, gene therapy expert and Principal Investigator of this FA study, who was available at the end of the presentation for medical/science related questions. AAVrh.10hFXN is an investigational therapy that is under evaluation and not approved by the FDA. Anyone considering participating in a clinical trial or trying a new medication should discuss the matter with their physician. The NAF does not endorse or recommend any particular studies or treatment options. For more information on Ataxia, please visit our website: https://www.ataxia.org Become a Free NAF member! https://bit.ly/JoinNAF Follow us on Social Media! NAF Facebook:   / ataxiafounda.  . NAF Twitter:   / naf_ataxia   NAF Instagram:   / ataxiafound.  . Ataxia Subreddit:   / ataxia   Speaker: Madeline Galbraith, BA Institution: Weill Cornell Medicine Bio: Madeline (Maddie) Galbraith is the research coordinator for Weill Cornell Medicine’s Department of Genetic Medicine, supporting various phase I gene therapy clinical trials. She primarily works on their Friedreich’s Ataxia study, in addition to studies for Alzheimer’s Disease and cocaine addiction. In her role, she manages research subject participation and study-related assessments, contributing to the overall clinical operations of the research program. As the direct point of contact for research subjects and their families, Maddie takes pride in building strong relationships with participants and strives to coordinate study visits with each family’s needs in mind. Maddie is grateful for her subjects’ tendency to explore NYC during study visits, leading to many new restaurant and activity recommendations for herself and other study participants! Before joining Weill Cornell, Maddie was a student at Claremont McKenna College, where she graduated magna cum laude with a Bachelor of Arts in Biology with honors. Maddie is a fan of the Eagles band and enjoys discussing rock music and current pop hits with Dr. Crystal. Speaker: Ronald G. Crystal, MD Institution: Weill Cornell Medicine Bio: Dr. Ronald G. Crystal is Professor and Chairman of the Department of Genetic Medicine at Weill Cornell Medicine, where he is also the Bruce Webster Professor of Internal Medicine, Director of the Belfer Gene Therapy Core Facility and Attending Physician at the NewYork-Presbyterian Hospital/Weill Cornell Medical Center. With 50 years of experience as a pulmonary physician, Dr. Crystal’s early research focused on lung diseases, particularly lung fibrosis and alpha 1-antitrypsin deficiency, for which he developed an FDA-approved therapy now used to treat thousands of patients worldwide. In the late 1980s, he became a pioneer in gene therapy and was the first to use a recombinant virus to treat a human genetic disorder. His work laid the foundation for adenovirus-based gene therapies and vaccines. He has since led numerous gene therapy clinical trials and is the Principal Investigator for several ongoing studies, including a phase I trial targeting the cardiomyopathy of Friedreich’s Ataxia. Before joining Weill Cornell, Dr. Crystal served as Chief of the Pulmonary Branch at the National Heart, Lung, and Blood Institute. He earned his MD from the University of Pennsylvania and completed post-graduate training at Massachusetts General Hospital and the University of California, San Francisco. A Fellow of the National Academy of Inventors, he has published over 900 scientific papers with 84,000+ citations, holds multiple biomedical patents, and has founded several biotechnology companies focused on gene therapy. Outside of his professional activities, Dr. Crystal is an active mountain climber and a jazz music enthusiast.