Results 171 to 180 of about 3,107 (205)

Safety of Onasemnogene Abeparvovec Administration to Type 1 SMA Patients Who Have Received Risdiplam

Journal of Clinical Neuromuscular Disease
Three therapies are now available for the treatment of type 1 spinal muscular atrophy: onasemnogene abeparvovec (OA), nusinersen, and risdiplam. We present a retrospective, single-center case series detailing our center's experience with six patients ...
Sivakami Chelladurai, Sarah D’Urso, Mark Atherton, Min Tsui Ong   +3 more
semanticscholar   +1 more source

Onasemnogene-abeparvovec

Reactions Weekly, 2022
  +6 more sources

[Onasemnogene abeparvovec gene replacement therapy for spinal muscular atrophy in children: a Moscow experience].

Zhurnal Nevrologii i Psikhiatrii imeni S.S. Korsakova
OBJECTIVE To assess the efficacy and safety of onasemnogene abeparvovec (OA) in children with spinal muscular atrophy (SMA) treated between 2021 and 2025 at a Moscow regional rare disease referral center (Morozov Children's City Clinical Hospital ...
V. S. Lobanova, Yu. E. Martynenko, A. V. Vlasova, I. Vitkovskaya, T. N. Kekeeva, N. L. Pechatnikova, A. A. Chudaykina, N. A. Krasnoschekova, A. A. Rahalina, V. Zarubina, Z. I. Esmurzieva, S. Valieva, V. V. Gorev   +12 more
semanticscholar   +1 more source

Three patients with spinal muscular atrophy who have switched treatments risdiplam following onasemnogene abeparvovec, case report

São Paulo Medical Journal
Introduction: 5q spinal muscular atrophy (SMA) is the most common motor neuron disease of childhood. Patients present with hypotonia, symmetrical proximal muscle weakness, and areflexia.
João Paulo Barile, W. B. Pinto, P. Serrano, I. B. Farias, Renan Brandão Rambaldi Cavalheiro, Patrícia Marques Mendes, Eduardo Mendonça Werneck da Silva, Daniel Delgado Seneor, Letícia Machado Dumont, Acary Souza Bulle, Paulo Sgobbi   +10 more
semanticscholar   +1 more source

Reduction of Preexisting AAV9 Antibody Titers Before Onasemnogene Abeparvovec Administration in Twins With Spinal Muscular Atrophy.

Neurology
OBJECTIVES Preexisting immunity to adeno-associated viruses (AAVs) presents a major obstacle to eligibility for gene therapy. METHODS We determined the feasibility of immunodepletion with therapeutic plasma exchange and rituximab to lower anti-AAV9 ...
Ryan W Nelson, Cody A Cox, Molly M Stark, Andrew D Johnson, Kelly Sichmeller, Jayne Earhart, J. M. Ihinger, Seth A. Stafki, Peter I Karachunski, Peter B. Kang   +9 more
semanticscholar   +1 more source

Onasemnogene abeparvovec gene therapy for treatment of patients with spinal muscular atrophy: Updated real-world practical considerations.

Journal of Neuromuscular Diseases
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease resulting from biallelic pathogenic variants of the survival motor neuron 1 (SMN1) gene that leads to motor neuron degeneration, progressive muscle atrophy, and weakness.
Crystal M Proud, Elizabeth A Kichula, Susan E. Matesanz, Ashutosh Kumar, Kayoko Saito, Chamindra G. Laverty, M. Farrar, Diana Bharucha‐Goebel, Jana Haberlová, Vivek Mundada, Jennifer M Kwon, Hugh J Mcmillan   +11 more
semanticscholar   +1 more source

Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy

CNS Drugs, 2022
Onasemnogene abeparvovec (Zolgensma®) is a gene therapy approved for the treatment of spinal muscular atrophy (SMA). Administered as a one-time intravenous infusion, onasemnogene abeparvovec uses the adeno-associated virus vector to deliver a functional copy of the human survival motor neuron (SMN) gene to motor neuron cells.
openaire   +2 more sources

Onasemnogene Abeparvovec-xioi: Gene Therapy for Spinal Muscular Atrophy

Annals of Pharmacotherapy, 2020
Objective: To review the efficacy and safety of onasemnogene abeparvovec-xioi (Zolgensma) in the treatment of spinal muscular atrophy (SMA). Data Sources: An English-language literature search of PubMed, MEDLINE, and Ovid (1946 to December 2019) was completed using the terms onasemnogene, AVXS-101, and spinal muscular atrophy.
Debra Stevens, Melanie K. Claborn, Brooke L. Gildon, Tiffany L. Kessler, Cheri Walker   +4 more
openaire   +2 more sources

Onasemnogene-abeparvovec

Reactions Weekly, 2021
  +4 more sources

Onasemnogene Abeparvovec: First Global Approval

Drugs, 2019
Onasemnogene abeparvovec (onasemnogene abeparvovec-xioi; formerly AVXS-101; ZOLGENSMA®) is an adeno-associated viral vector-based gene therapy designed to deliver a functional copy of the human survival motor neuron (SMN) gene to the motor neuron cells of patients with spinal muscular atrophy (SMA).
openaire   +2 more sources