Presented by Elizabeth Berry-Kravis, MD, PhD, Craig A. Erickson, MD, and Randi J. Hagerman, MD
This session will be a family-friendly joint presentation of results from recently completed, and a description of all currently active clinical trials and development programs for new medications in fragile X syndrome (FXS). Trials to be covered include acamprosate, single dose EEG-focused challenge studies, AFQ056 (FXLEARN), ergoloid mesylates/5HTP, metformin, cannabidiol, BAER-101, BPN 14770, sulindac and Anavex 2-73. A basic explanation of how each medication works and the results of trials previously conducted will be discussed. For each trial currently recruiting, requirements for participation, sites where patients can participate, and logistical information will be given, and presenters will answer any questions about trials.
Runtime: 1:18:09
Additional resources and controls for this video are accessible just below the video: Play/pause, volume, subtitles, view transcript, watch as picture-in-picture, or in fullscreen mode.
About the Speakers
Elizabeth Berry-Kravis, MD, PhD
Rush University Medical Center
Professor of Pediatrics, Neurological Sciences, and Biochemistry
Dr. Elizabeth Berry-Kravis is a professor of pediatrics, neurological sciences, and biochemistry at Rush University Medical Center in Chicago. She attended the University of Notre Dame for her undergraduate studies and the University of Chicago for her medical degree, PhD, and training in pediatric neurology. She moved to Rush in 1992 and established the Fragile X Clinic and Research Program, through which she provides care to over 700 patients with Fragile X syndrome. She has studied Fragile X syndrome medical issues, epilepsy, and psychopharmacology, and has been a leader in translational research, including the development of outcome measures and biomarkers, natural history studies, newborn screening, and particularly clinical trials of new targeted treatments. Her laboratory studies the cellular roles of fragile X mental retardation protein (FMRP), its relationship to phenotypes, and the optimization of genetic testing methods.
Craig Erickson, MD
Cincinnati Children’s Hospital Medical Center
Professor of Psychiatry
Dr. Craig A. Erickson is a professor of psychiatry at Cincinnati Children’s Hospital Medical Center and the University of Cincinnati College of Medicine-Affiliated. Dr. Erickson leads a neurodevelopmental clinical and research group focused on improving clinical care through research discovery. He is the Director of the Cincinnati Fragile X Research and Treatment Center which is one of the largest such programs in the world. He serves as the Chair of the Clinical Trials Committee organized by the National Fragile X Foundation and is a leader in translational medicine efforts in fragile X syndrome, autism, and related disorders. Additionally, he is the director of research in the Division of Psychiatry at Cincinnati Children’s Hospital.
Randi J. Hagerman, MD
MIND Institute, Fragile X Research and Treatment Center
Medical Director
Dr. Randi Hagerman is a developmental and behavioral pediatrician who has worked in the Fragile X field for over 30 years on both FXS and premutation disorders. She is at the MIND Institute at UC Davis where she holds an Endowed Chair in Fragile X Research and she runs the Clinical Trials Program and is the medical director of the MIND Institute. She has published over 400 articles related to Fragile X and related disorders. In the spirit of mentorship and collaboration, the NFXF Summer Scholar Program proudly bears Dr. Hagerman’s name and is now the Randi J. Hagerman Summer Scholars Research Award.
Hilary Rosselot
National Fragile X Foundation
Executive Director, Research Facilitation Lead
Hilary is the executive director of the National Fragile X Foundation and leads the Research Facilitation programming. Before joining the NFXF team, she worked at the Cincinnati Fragile X Research and Treatment Center for over 5 years. She has experience as a Clinical Research Coordinator across many types of clinical trials and served as the Clinical Research Manager for the Cincinnati program.
Additional Resources
Fragile X and Puberty & Sexuality
Depending on their age, it’s necessary to approach teaching children about relationships, the difference between public and private, body changes, feelings and emotions, reproduction, STDs, birth control, and how to keep themselves safe. You may even find you need a refresher for yourself before approaching the subject with them.