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Regulatory: The Future Pathway to Access

Monday, November 6, 2023 at 12:30 pm US EDT -

Join us for an informative and engaging webinar where we delve into the complex regulatory aspects of individualized medicines in the United States and Europe. A discussion panel will shed light on the evolving landscape and discuss how we can collectively shape the future of these critical endeavors, driving innovation and streamlining approval processes.

Prof. Dr. Annemieke Aartsma-Rus is a professor of Translational Genetics at the Department of Human Genetics of the Leiden University Medical Center. She played an important role in the development of antisense mediated exon skipping for Duchenne muscular dystrophy during her PhD research (2000-2004) at the Leiden University Medical Center (the Netherlands). As of December 2007 she became leader of the “DMD exon skip group”. Since 2013 she has a visiting professorship at the Institute of Genetic Medicine of Newcastle University (UK). In 2020 she co-founded the Dutch Center for RNA Therapeutics (DCRT), a not-for-profit academic collaboration aiming to develop clinical treatment with exon skipping therapies for eligible patients with unique mutations.

Dr. Yu completed his undergraduate degree in biochemistry and molecular biology at Harvard College, his M.D. and Ph.D. degree (in Neuroscience) from the University of California at San Francisco, clinical neurology training at Massachusetts General Hospital and Brigham and Women’s Hospital, and a fellowship in neurodevelopmental genetics at MGH and Boston Children’s Hospital. He joined the faculty in the Division of Genetics and Genomics as Instructor in 2010 and Associate Professor in 2013.

Dr. Balabanov is a clinical neurologist who before joining the EMA worked as a consultant neurologist at the Clinic of Neurology, University Hospital – Plovdiv, Bulgaria. Dr. Balabanov has a PhD in Neurology and holds a position of senior teaching professor of neurology at the Department of Neurology, Medical University of Plovdiv.

Currently Dr. Balabanov leads the office for Therapies for Neurological and Psychiatric Disorders at the EMA.

In his career at the EMA, Dr. Balabanov has been involved in the development of the regulatory guidelines for development of new drugs in the field of Multiple sclerosis, Duchenne Muscular Dystrophy (DMD) and Amyotrophic Lateral Sclerosis (ALS).

In the spirit of enhancing stakeholder collaboration Dr. Balabanov was responsible for a number of scientific workshops at the EMA dealing with:

• The future of the development of new drugs in MS

• Issues in the development of therapies in Duchenne Muscular Dystrophy

• Clinical Trial Readiness in Spinal Muscular Atrophy (SMA)

• Registries in MS and their role in drug evaluation.

Dr. Balabanov has significant experience in process development and implementation as the lead of a task force introducing improved operating procedures in the management of new marketing authorization applications.

Previously at the EMA Dr. Balabanov worked as a Product lead for Neurology medicinal products, and was a member of the Scientific Advice section of the Agency, providing advice to applicants, aimed at improving their drug development programmes and making them adherent to the current regulatory and scientific requirements.

Emily Freilich, MD, is the Division Director of the Division of Neurology 1 within the Office of New Drugs in FDA’s Center for Drug Evaluation and Research. Dr. Freilich is a board-certified pediatric neurologist. Dr. Freilich graduated from Duke University with a Bachelor of Science degree in biology, and received her medical degree from Rutgers-New Jersey Medical School. She completed her pediatric residency and child neurology training at Children’s National Health System in Washington, D.C. Prior to joining FDA, Dr. Freilich worked at Children’s National and the Pediatric Specialists of Virginia, where she was a general child neurologist with special interest in rare pediatric epilepsies, and served as co-director of the Tuberous Sclerosis Clinic. Dr. Freilich was on the medical staff at Children’s National and Inova Fairfax Hospitals, and was an Assistant Professor of Pediatrics and Neurology at George Washington University School of Medicine, where she was involved in the education of medical students, pediatric residents, and neurology fellows. She joined the Division of Neurology Products at FDA in 2016, where she worked as a clinical reviewer and team leader in the areas of epilepsy, migraine, neuromuscular, and rare neurologic disorders. She subsequently served as Acting Associate Director for Therapeutic Review and then Acting Deputy Director in the Division of Neurology 1 prior to her current role.



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