Trusted Resources: Education
Scientific literature and patient education texts
Gene Replacement Therapy With Onasemnogene Abeparvovec in Children With Spinal Muscular Atrophy Aged 24 Months or Younger and Bodyweight up to 15 Kg: An Observational Cohort Study
source: The Lancet. Child & Adolescent Health
authors: Weiß C,Ziegler A,Becker LL,Johannsen J,Brennenstuhl H,Schreiber G,Flotats-Bastardas M,Stoltenburg C,Hartmann H,Illsinger S,Denecke J,Pechmann A,Müller-Felber W,Vill K,Blaschek A,Smitka M,van der Stam L,Weiss K,Winter B,Goldhahn K,Plecko B,Horber V,Bernert G,Husain RA,Rauscher C,Trollmann R,Garbade SF,Hahn A,von der Hagen M,Kaindl AMsummary/abstract:
Given the novelty of gene replacement therapy with onasemnogene abeparvovec in spinal muscular atrophy, efficacy and safety data are limited, especially for children older than 24 months, those weighing more than 8·5 kg, and those who have received nusinersen. We aimed to provide real-world data on motor function and safety after gene replacement therapy in different patient subgroups.organization: Department of Pediatric Neurology, Charité-Universitätsmedizin Berlin, Berlin, Germany; Center for Chronically Sick Children, Charité-Universitätsmedizin Berlin, Berlin, Germany.
The Gwendolyn Strong FoundationAt Gwendolyn Strong Foundation, we work ...
Jerry R. Mendell, MDJerry R. Mendell, MD, is an Attending Ne...
Patient of the Month: GiannaAfter noticing that her daughter was una...
Spinal Muscular Atrophy: From Rags to RichesThe aim of this paper is to provide a sh...
SMA Gene Replacement Therapyhttps://www.youtube.com/watch?v=vHbJs8bm...
Infants With Symptomatic SMA Reach Milestones With Gene TherapyGene replacement therapy with onasemnoge...
Pediatric Spinal Muscular AtrophyWhat is spinal muscular atrophy (SMA)?Sp...