Results 151 to 160 of about 3,107 (205)

AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort

, 2019
Samiah Al-Zaidy, Stephen J. Kolb, Linda Lowes, Lindsay N. Alfano, Richard Shell, Kathleen R. Church, Sukumar Nagendran, Douglas M. Sproule, Douglas E. Feltner, Courtney Wells, Francis G. Ogrinc, Melissa Menier, James L’Italien, W. David Arnold, John T. Kissel, Brian K. Kaspar, Jerry R. Mendell   +16 more
openalex   +2 more sources

Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings. [PDF]

Gene Ther, 2023
Chand DH, Sun R, Diab KA, Kenny D, Tukov FF.   +4 more
europepmc   +1 more source

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG). [PDF]

J Neuromuscul Dis, 2023
Finkel RS, Darras BT, Mendell JR, Day JW, Kuntz NL, Connolly AM, Zaidman CM, Crawford TO, Butterfield RJ, Shieh PB, Tennekoon G, Brandsema JF, Iannaccone ST, Shoffner J, Kavanagh S, Macek TA, Tauscher-Wisniewski S.   +16 more
europepmc   +1 more source

Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. [PDF]

Drug Saf, 2022
Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH.   +9 more
europepmc   +1 more source

Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study. [PDF]

Gene Ther, 2023
Bitetti I, Lanzara V, Margiotta G, Varone A.   +3 more
europepmc   +1 more source

Characteristics of Medical Products Comprising Human Cells, Genes, or Tissues Developed in Japan and the European Union Compared via Public Assessment Reports [PDF]

, 2020
Ito, Tatsuya, Kasai, Hiroi, Kurauchi, Ryosuke   +2 more
core   +1 more source

Acute liver failure worsened after respiratory syncytial virus infection in an infant with spinal muscular atrophy type I after receiving onasemnogene abeparvovec

Shohei Sakemi, Takako Fujita, Noriyuki Kaku, Shuichi Yatsuga, Kazutoshi Ito, Daiki Sasaoka, Hiromi Yamaguchi, Hitomi Hayashi, Takahito Inoue, Kanako Higashi, Yasunari Sakai, Shouichi Ohga, Shinichiro Nagamitsu   +12 more
openalex   +1 more source

Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study. [PDF]

Lancet Reg Health Eur
Weiß C, Becker LL, Friese J, Blaschek A, Hahn A, Illsinger S, Schwartz O, Bernert G, Hagen MV, Husain RA, Goldhahn K, Kirschner J, Pechmann A, Flotats-Bastardas M, Schreiber G, Schara U, Plecko B, Trollmann R, Horber V, Wilichowski E, Baumann M, Klein A, Eisenkölbl A, Köhler C, Stettner GM, Cirak S, Hasselmann O, Kaindl AM, Garbade SF, Johannsen J, Ziegler A, SMArtCARE and Swiss-Reg-NMD study group.   +31 more
europepmc   +1 more source

Table 3_Real-world outcomes of spinal muscular atrophy treatment with onasemnogene abeparvovec in Croatia: a comprehensive case series and literature review.docx

Ivan Lehman (21590618), Matija Matošević (14619110), Lovro Lamot (674823), Branka Bunoza (21590621)   +3 more
openalex   +1 more source