美国食品和药物管理局以患者为中心的药物开发指南:在罕见的发育和性脑病变中对试验准备的考虑

Anne T Berg¹, Natasha N Ludwig¹, Mary Wojnaroski¹+5

• ¹From the Department of Neurology (A.T.B.), Northwestern-Feinberg School of Medicine, Chicago, IL; Decoding Developmental Epilepsies (A.T.B., G.C., J.Z.H.), Washington, DC; Department of Neuropsychology (N.N.L.), Kennedy Krieger Institute; Department of Psychiatry and Behavioral Sciences (N.N.L.), The Johns Hopkins School of Medicine, Baltimore, MD; Department of Psychology (M.W.), Nationwide Children's Hospital; Department of Pediatrics (M.W.), The Ohio State University, Columbus; Ardea Outcomes (C.A.T.C.), Halifax, Nova Scotia, Canada; Connections Beyond Sight and Sound Maryland & DC Deaf-Blind Project (R.H.), University of Maryland, College Park; The Inchstone Project (J.Z.H.); Telethon Kids Institute (J.D.), The University of Western Australia; and Curtin School of Allied Health (J.D.), Curtin University, Perth, Western Australia.