Results 141 to 150 of about 2,700 (197)

Administration of Onasemnogene Abeparvovec in an Infant With Spinal Muscular Atrophy and PCR-Confirmed SARS-CoV-2 Infection. [PDF]

Cureus
Shimomura I, Maruyama S, Kodama Y, Matsunaga M, Okamoto Y.   +4 more
europepmc   +1 more source

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

Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial. [PDF]

Nat Med
Proud CM, Vũ DC, Wilmshurst JM, Sanmaneechai O, Gulati S, Xiong H, Moreno HC, Tay SKH, Thong MK, Born AP, Banzzatto Ortega A, Jong YJ, Al-Muhaizea MA, Lee AW, Visootsak J, Tauscher-Wisniewski S, Alecu I, Parlikar R, Finkel RS, STEER Study Group.   +19 more
europepmc   +1 more source

Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment. [PDF]

J Neuromuscul Dis, 2023
McGrattan KE, Shell RD, Hurst-Davis R, Young SD, O'Brien E, Lavrov A, Wallach S, LaMarca N, Reyna SP, Darras BT.   +9 more
europepmc   +1 more source

Evaluation of cardiac function in patients with SMA after treatment with onasemnogene abeparvovec [PDF]

Akihisa Horigome, Satoshi Takasago, Reiko Arakawa, Katsuyoshi Shimozawa, Masao Kaneshige, Keiji Goishi, Hideko Uryu, Junko Yamanaka, Hiroyuki Shichino, Ayumi Mizukami   +9 more
openalex   +1 more source

Switching therapies: safety profile of Onasemnogene abeparvovec-xioi in a SMA1 patient previously treated with Risdiplam. [PDF]

Acta Myol, 2022
Tosi M, Catteruccia M, Cherchi C, Mizzoni I, D'Amico A.   +4 more
europepmc   +1 more source

Fatal outcomes following onasemnogene abeparvovec in advanced-stage spinal muscular atrophy. [PDF]

Gene Ther
Pongsakornkullachart P, Kulsirichawaroj P, Kongkasuwan R, Tovichien P, Jitwongwai S, Kanjanauthai S, Preeprem N, Limpaninlachat S, Sermpon N, Sanmaneechai O.   +9 more
europepmc   +1 more source

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

Onasemnogene Abeparvovec Treatment after Nusinersen in an Infant with Spinal Muscular Atrophy Type 1

, 2022
DAIKI NANRI, Kotaro Yuge, KOHEI GOTO, Takuro Kimura, Yukako Yae, Tatsuki Mizuochi, Ryosuke Sato, Tomoyo Itonaga, Tomoki Maeda, Yushiro Yamashita   +9 more
openalex   +2 more sources

Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy. [PDF]

J Clin Invest
Proud CM, Finkel RS, Parsons JA, Masson R, Brandsema JF, Kuntz NL, Foster R, Li W, Littauer R, Sohn J, Fradette S, Youn B, Paradis AD, RESPOND Study Group.   +13 more
europepmc   +1 more source