Results 151 to 160 of about 2,700 (197)

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

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

Safety and tolerability of onasemnogene abeparvovec for patients with spinal muscular atrophy weighing ≤17 kg and ≤24 months old from OFELIA, a phase 4, open-label, multicenter, non-randomised, interventional study. [PDF]

Lancet Reg Health Am
Saute JA, Muntadas J, Gurgel-Giannetti J, Monges S, Aliberti P, Mendonça RH, Alecu I, Ritter S, Martins de Lana J, Mumneh N, Zanoteli E, OFELIA Study Group.   +11 more
europepmc   +1 more source

Trach and treat: Safety and motor outcomes following onasemnogene abeparvovec in patients with spinal muscular atrophy and tracheostomies in the RESTORE registry

Yasemin Erbaş, Laurent Servais, Perry B. Shieh, Natalie L. Goedeker, Megan A. Waldrop, Ryosuke Bo, Dheeraj Raju, Kamal Benguerba, Sandra P. Reyna, David Wolff, Richard S. Finkel   +10 more
openalex   +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

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

Level of circulating neurofilaments as a biomarker of spinal muscular atrophy during gene therapy with onasemnogene abeparvovec

Daria A. Chudakova, Eugeniya V. Uvakina
openalex   +2 more sources

Emerging thrombotic disorders associated with virus-based innovative therapies: from VITT to AAV-gene therapy-related thrombotic microangiopathy [PDF]

Benemei, Silvia, Marcucci, Rossella
core   +1 more source