Cargando…

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

Spinal muscular atrophy (SMA) is a genetically inherited recessive neuromuscular disease that causes muscular atrophy and weakness. Onasemnogene abeparvovec (formerly AVXS-101, Zolgensma®, Novartis) is a targeted therapy approved to treat patients with SMA in >40 countries worldwide. This study d...

Descripción completa

Detalles Bibliográficos
Autores principales:	Bitetti, Ilaria, Lanzara, Valentina, Margiotta, Giovanna, Varone, Antonio
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2022
Materias:	Brief Communication
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10457192/ https://www.ncbi.nlm.nih.gov/pubmed/35606491 http://dx.doi.org/10.1038/s41434-022-00341-6

Ejemplares similares

Onasemnogene abeparvovec for spinal muscular atrophy
Publicado: (2022)

Onasemnogene Abeparvovec for Spinal Muscular Atrophy: The Costlier Drug Ever
por: Mahajan, Rajiv
Publicado: (2019)

Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy
por: D'Silva, Arlene M., et al.
Publicado: (2022)

Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies
por: Pane, Marika, et al.
Publicado: (2023)

Combination Therapy with Nusinersen and Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy Type I
por: Mirea, Andrada, et al.
Publicado: (2021)

Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study
por: Stettner, Georg M., et al.
Publicado: (2023)

Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy
por: Kichula, Elizabeth A., et al.
Publicado: (2021)

Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy
por: René, Charlotte A., et al.
Publicado: (2023)

Safety Monitoring of Gene Therapy for Spinal Muscular Atrophy with Onasemnogene Abeparvovec –A Single Centre Experience
por: Friese, Johannes, et al.
Publicado: (2021)

Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy
por: Mendell, Jerry R., et al.
Publicado: (2021)

Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma)
por: Ogbonmide, Tolu, et al.
Publicado: (2023)

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)
por: Finkel, Richard S., et al.
Publicado: (2023)

Erratum to: Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment
por: McGrattan, Katlyn E., et al.
Publicado: (2023)

Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment(1)
por: McGrattan, Katlyn E., et al.
Publicado: (2023)

Early Development of Spinal Deformities in Children Severely Affected with Spinal Muscular Atrophy after Gene Therapy with Onasemnogene Abeparvovec—Preliminary Results
por: Soini, Venla, et al.
Publicado: (2023)

Changes in electrophysiological findings of spinal muscular atrophy type I after the administration of nusinersen and onasemnogene abeparvovec: two case reports
por: Mizuno, Tomoko, et al.
Publicado: (2023)

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy
por: Day, John W., et al.
Publicado: (2021)

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial
por: Strauss, Kevin A., et al.
Publicado: (2022)

Correction to: Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy
por: Day, John W., et al.
Publicado: (2021)

An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)
por: Dean, Rebecca, et al.
Publicado: (2021)

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial
por: Strauss, Kevin A., et al.
Publicado: (2022)

Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings
por: Chand, Deepa H., et al.
Publicado: (2023)

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec
por: Day, John W., et al.
Publicado: (2021)

Ultraexpensive gene therapies, industry interests and the right to health: the case of onasemnogene abeparvovec in Brazil
por: Ivama-Brummell, Adriana Mitsue, et al.
Publicado: (2022)

Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys
por: Tukov, Francis Fonyuy, et al.
Publicado: (2022)

Switching therapies: safety profile of Onasemnogene abeparvovec-xioi in a SMA1 patient previously treated with Risdiplam
por: Tosi, Michele, et al.
Publicado: (2022)

Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey
por: Toro, Walter, et al.
Publicado: (2023)

Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients
por: Malone, Daniel C., et al.
Publicado: (2019)

A National Spinal Muscular Atrophy Registry for Real-World Evidence
por: Hodgkinson, Victoria L., et al.
Publicado: (2020)

Real-World Data on Access to Standards of Care for People With Spinal Muscular Atrophy in the UK
por: Muni-Lofra, Robert, et al.
Publicado: (2022)

Significant healthcare burden and life cost of spinal muscular atrophy: real-world data
por: Chan, Sophelia H. S., et al.
Publicado: (2022)

Clinical Characteristics of Spinal Muscular Atrophy in Korea Confirmed by Genetic Analysis
por: Hwang, Heewon, et al.
Publicado: (2017)

Mitochondrial Dysfunction in Spinal Muscular Atrophy
por: Zilio, Eleonora, et al.
Publicado: (2022)

Spinal muscular atrophy
por: D'Amico, Adele, et al.
Publicado: (2011)

Carrier Frequency of Spinal Muscular Atrophy in a Large-scale Korean Population
por: Park, Jong Eun, et al.
Publicado: (2020)

Increased systemic HSP70B levels in spinal muscular atrophy infants
por: Eichelberger, Eric J., et al.
Publicado: (2021)

Combination disease‐modifying treatment in spinal muscular atrophy: A proposed classification
por: Proud, Crystal M., et al.
Publicado: (2023)

Low fat diets increase survival of a mouse model of spinal muscular atrophy
por: Deguise, Marc‐Olivier, et al.
Publicado: (2019)

Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy
por: Pane, Marika, et al.
Publicado: (2022)

Effectiveness of Nusinersen in Type 1, 2 and 3 Spinal Muscular Atrophy: Croatian Real-World Data
por: Belančić, Andrej, et al.
Publicado: (2023)

Cannot write session to /tmp/vufind_sessions/sess_ffj6fohkec9qrsbo8vo5id5go6