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Scholar Rock SMA KOL Event

Start Date:6/15/2021

Start Time:10:00 AM EDT

Duration:90 minutes


Scholar Rock is pleased to host a KOL event and panel discussion on the therapeutic potential of apitegromab, a selective inhibitor of myostatin activation, in Spinal Muscular Atrophy (SMA). Positive results from the 12-month TOPAZ Phase 2 trial (NCT03921528) that evaluated apitegromab in patients with Type 2 and 3 SMA were announced in early April. Dr. Crawford will be presenting at the 2021 Virtual SMA Research and Clinical Care Meeting, being held in conjunction with Cure SMA’s Annual SMA Conference on June 11, 2021. 

Over the course of this event, we will discuss the results of the TOPAZ trial, the unmet medical needs of individuals with SMA, as well as host a panel discussion with two investigators from the TOPAZ trial, Thomas Crawford, M.D. of Johns Hopkins Medicine and Basil Darras, M.D. of Boston Children’s Hospital.

If you're already registered for the Scholar Rock SMA KOL Event webcast, click below:



Tony Kingsley

President & CEO

Scholar Rock

Tony Kingsley has served as President & CEO since August 2020. He was most recently the president and chief executive officer of TARIS® Bio, an oncology company that was acquired by Janssen Pharmaceuticals in late 2019. Prior to TARIS, Mr. Kingsley held a series of leadership roles in the life sciences industry, including president and chief operating officer of The Medicines Company, the head of global commercial at Biogen, and a division president at Hologic, Inc. and Cytyc Corp. Mr. Kingsley was a partner at McKinsey & Company based in New York and Boston. He received a BA in government from Dartmouth College and an MBA from Harvard Business School.

Yung Chyung, MD

Chief Medical Officer

Scholar Rock

Dr. Yung H. Chyung has served as Chief Medical Officer since February 2016 and leads the clinical development of pipeline candidates and medical affairs. Prior to joining Scholar Rock, Yung served in roles of increasing responsibility at Dyax Corp. (acquired by Shire Plc in January 2016) from 2011 to February 2016, most recently serving as Vice President of Medical Research, where he was responsible for clinical research and medical affairs. From 2010 to 2011, he worked at Genzyme Corporation where he was responsible for medical affairs efforts globally for multiple rare disease programs. Yung earned his MD from Harvard Medical School and completed his internal medicine residency and allergy and immunology fellowship at Massachusetts General Hospital. He holds an AB in Biochemical Sciences from Harvard College.

Thomas Crawford, MD

Lead TOPAZ Principal Investigator; Co-Director of MDA Clinic, John Hopkins Medicine

Dr. Tom Crawford has been a member of the Department of Neurology at Johns Hopkins since 1987. He is co-director of the MDA clinic for Neuromuscular Disorders and Neurologist for the Ataxia Telangiectasia Clinical Center at Johns Hopkins. His practice involves general child neurology with a principal interest in caring for children with neuromuscular, neuromotor, and ataxic disorders. Dr. Crawford received his medical degree from the University of Southern California. He completed a pediatric internship and residency at the Pediatric Pavilion of the Los Angeles County / University of Southern California Medical Center, followed by a pediatric chief residency. He completed his training in Neurology with Special Qualification in Child Neurology at the Los Angeles Children's Hospital.

Basil Darras, MD

TOPAZ Trial Investigator; Associate Neurologist-in-Chief at Boston Children’s Hospital

Dr. Darras serves as an expert for the Department of Neurology for Boston Children's Hospital Precision Medicine Service. He is the associate neurologist-in-Chief at Boston Children's Hospital and holds the Joseph J. Volpe Chair in Neurology at Harvard Medical School. He is also the Chief of the division of clinical neurology in the Department of Neurology at the hospital. His special focus is in the care of children with neuromuscular conditions originating from inherited or acquired conditions of the motor unit. Following medical school in Greece and residencies in pediatrics and child neurology at the State University of New York at Stony Brook and Tufts Medical Center, respectively, he completed a post-doctoral fellowship at Yale in genetics.

Jill Jarecki, PhD

Chief Scientific Officer, CureSMA

Dr. Jarecki manages a broad portfolio of SMA research investments in basic research, preclinical drug development, and clinical trial networks, which has facilitated the FDA approval of three therapies. More recently, she has been engaged in patient focused drug development, by forming an industry collaboration of biotech / pharmaceutical partners, to jointly advance drug development activities. Dr. Jarecki received her PhD in genetics from Yale University and conducted postdoctoral research at Stanford University. Prior to Cure SMA, she spent 5 years working in the biotech industry at Vertex Pharmaceuticals and Invitrogen Corporation.
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