Results 31 to 40 of about 2,067 (154)

Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study. [PDF]

Neurology
BACKGROUND AND OBJECTIVES: Safety and efficacy of IV onasemnogene abeparvovec has been demonstrated for patients with spinal muscular atrophy (SMA) weighing \u3c 8.5 kg.
McMillan HJ, Baranello G, Farrar MA, Zaidman CM, Moreno T, De Waele L, Jong YJ, Laugel V, Quijano-Roy S, Mercuri E, Chien YH, Straub V, Darras BT, Seibert J, Bernardo Escudero R, Alecu I, Freischläger F, Muntoni F, SMART Study Group.   +18 more
europepmc   +2 more sources

Is gene therapy in spinal muscular atrophy safe? A casereport of thrombotic microangiopathy following onasemnogene abeparvovec

Journal of Pre-Clinical and Clinical Research, 2022
Spinal muscular atrophy is a neuromuscular disorder caused by a mutation in the survival of SMN1 gene. Diagnosis of the disease is based mainly on the presence of hypotonia and symmetrical [1].
Aleksandra Ziółkiewicz, Michał Bielak, Iwona Welian, Magdalena Chrościńska- Krawczyk   +3 more
doaj   +1 more source

Interim Analysis of Treatment Outcomes of Young Children with 5q Spinal Muscular Atrophy on Gene Replacement Therapy with Onasemnogene Abeparvovec. Clinical Observations

Вопросы современной педиатрии, 2023
Background. Onasemnogene abeparvovec is the first gene replacement therapy medication based on the adeno-associated viral vector (AAV9). One injection to a patient with 5q spinal muscular atrophy (SMA) leads to replacement of the missing or defective ...
Anna A. Kokorina, Sergei S. Nikitin
doaj   +1 more source

Gene therapy for spinal muscular atrophy is considerably effective when administered as early as possible after birth

Molecular Genetics and Metabolism Reports, 2023
Introduction: Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by muscle atrophy and progressive muscle weakness. Insurance-approved treatments in Japan include antisense oligonucleotide therapy, gene therapy, and small molecule ...
Takaaki Sawada, Jun Kido, Yukako Yae, Kotaro Yuge, Keiko Nomura, Kentaro Okada, Natsumi Fujiyama, Shiro Ozasa, Kimitoshi Nakamura   +8 more
doaj   +1 more source

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy [PDF]

, 2022
INTRODUCTION: This is the first description of safety data for intravenous onasemnogene abeparvovec, the only approved systemically administered gene-replacement therapy for spinal muscular atrophy.
Tauscher-Wisniewski, Sitra, Reyna, Sandra P., Mendell, Jerry R., Day, John W., Finkel, Richard S., Strauss, Kevin A, Kleyn, Aaron, Chand, Deepa H., Tukov, Francis Fonyuy, Mercuri, Eugenio   +9 more
core   +1 more source

RWD129 Cost Effectiveness of Onasemnogene Abeparvovec in Infants with Presymptomatic Spinal Muscular Atrophy in Italy

, 2023
Objectives Spinal muscular atrophy (SMA) is a devastating rare disease and the most common genetic cause of infant death. Patients with SMA are lacking survival motor neuron 1 (SMN1) gene, leading to reduced SMN protein, loss of functional motor neurons,
Valentini, I.
core   +1 more source

Spinal muscular atrophy - onasemnogene abeparvovec and other therapeutic options [PDF]

Farmacja Polska, 2020
Spinal muscular atrophy (SMA) is a neuromuscular disorder that results in the loss of motor neurons. SMA is caused by mutations in the SMN1 gene, leading to the decreased synthesis of the SMN protein, necessary for motor neuron survival. In the past, SMA
Aleksandra Alicja Majchrzak-Celińska, Anna Warych, Mikołaj Szoszkiewicz   +2 more
doaj   +1 more source

Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments

Orphanet Journal of Rare Diseases, 2021
Background Spinal muscular atrophy (SMA) is a rare and devastating condition for which new disease-modifying treatments have recently been approved. Given the increasing importance of economic considerations in healthcare decision-making, this review ...
Tamara Dangouloff, Camille Botty, Charlotte Beaudart, Laurent Servais, Mickaël Hiligsmann   +4 more
doaj   +1 more source

Gene therapy-based strategies for spinal muscular atrophy—an Asia-Pacific perspective

Molecular and Cellular Pediatrics, 2023
Onasemnogene abeparvovec has been life-changing for children with spinal muscular atrophy (SMA), signifying the potential and progress occurring in gene- and cell-based therapies for rare genetic diseases.
Michelle A. Farrar, Loudella Calotes-Castillo, Ranil De Silva, Peter Barclay, Lani Attwood, Julie Cini, Monica Ferrie, Didu S. Kariyawasam   +7 more
doaj   +1 more source

Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry. [PDF]

J Neuromuscul Dis
International audience© 2024 - The authors. Published by IOS Press.Background: Long-term, real-world effectiveness and safety data of disease-modifying treatments for spinal muscular atrophy (SMA) are important for assessing outcomes and providing ...
Servais L, Day JW, De Vivo DC, Kirschner J, Mercuri E, Muntoni F, Proud CM, Shieh PB, Tizzano EF, Quijano-Roy S, Desguerre I, Saito K, Faulkner E, Benguerba KM, Raju D, LaMarca N, Sun R, Anderson FA, Finkel RS.   +18 more
europepmc   +8 more sources