WORLDSymposium 2022 – Avalglucosidase alfa Trial Data

Dr. Priya Kishnani, MD, Chen Family Professor of Pediatrics, Chief of the Division of Medical Genetics at Duke University Medical Center and Medical Director of the YT and Alice Chen Pediatrics Genetics and Genomics Center discusses recent data on Sanofi‘s Nexviazyme ® (avalglucosidase alfa) that was presented at 18th annual WORLDSymposium™ which showed sustained improvements in respiratory function and mobility in patients with Pompe disease.   

Dr. Kishnani is the C.L. and Su Chen Professor of Pediatrics; Medical Director, YT and Alice Chen Pediatrics Genetics and Genomics Center; and Division Chief, Medical Genetics, Duke University Medical Center. The center focuses on developing new therapies for rare genetic disorders.  She also serves as director of the lysosomal storage disease program, biochemical genetics training program, and metabolic clinic at DUMC.  Dr. Kishnani moved to the United States in 1991 after completing a residency in pediatrics in Mumbai, India. She completed a fellowship in clinical and biochemical genetics at Duke University Medical Center (DUMC) in 1995 and shortly thereafter joined the faculty at Duke University.  Dr. Kishnani is certified by the American Board of Medical Genetics and the American Board of Biochemical Genetics.  Dr. Kishnani’s work applies basic science discoveries to clinical trials and pioneering approval of new therapies. Her innovative contributions translate laboratory science into treatment for people with lysosomal storage disorders (LSDs), glycogen storage diseases (GSDs), Down syndrome and other inherited disorders.  Dr. Kishnani has been a lead investigator on several clinical trials for developing new drugs involving enzyme replacement therapy and small molecules. Her work is widely published in scientific journals and textbooks on areas including treatment, long-term complications and clinical trial results.  The care, treatment and natural history of individuals with LSDs, remain Dr. Kishnani’s passions. She emphasizes continuous care for chronic conditions using a team approach, consulting U.S. and international patients as well as medical colleagues in India.