Cargando…

Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment(1)

BACKGROUND: Improvement and maintenance of bulbar function are goals of disease-modifying treatments for spinal muscular atrophy (SMA). Lack of standardized measures and a widely accepted definition of bulbar function represents a gap in SMA care. OBJECTIVE: A multidisciplinary team conducted post-h...

Descripción completa

Detalles Bibliográficos
Autores principales:	McGrattan, Katlyn E., Shell, Richard D., Hurst-Davis, Rebecca, Young, Sally Dunaway, O’Brien, Eamonn, Lavrov, Arseniy, Wallach, Shiri, LaMarca, Nicole, Reyna, Sandra P., Darras, Basil T.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	IOS Press 2023
Materias:	Research Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10357176/ https://www.ncbi.nlm.nih.gov/pubmed/37092232 http://dx.doi.org/10.3233/JND-221531

Ejemplares similares

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)

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)

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)

Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes
por: Dunaway Young, Sally, 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)

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)

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

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)

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

Patient and Caregiver Outcomes After Onasemnogene Abeparvovec Treatment: Findings from the Cure SMA 2021 Membership Survey
por: Toro, Walter, 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)

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

Dysphagia Phenotypes in Spinal Muscular Atrophy: The Past, Present, and Promise for the Future
por: McGrattan, Katlyn Elizabeth, et al.
Publicado: (2021)

Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study
por: Bitetti, Ilaria, 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)

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)

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)

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)

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)

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)

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)

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

Development of an International SMA Bulbar Assessment for Inter-professional Administration
por: Dunaway Young, Sally, et al.
Publicado: (2023)

Androgens in spinal and bulbar muscular atrophy
Publicado: (2012)

Mitochondrial abnormalities in spinal and bulbar muscular atrophy
por: Ranganathan, Srikanth, et al.
Publicado: (2009)

Clinical features of spinal and bulbar muscular atrophy
por: Rhodes, Lindsay E., et al.
Publicado: (2009)

Swallowing markers in spinal and bulbar muscular atrophy
por: Banno, Haruhiko, et al.
Publicado: (2017)

The role of ubiquitination in spinal and bulbar muscular atrophy
por: Sengupta, Medha, et al.
Publicado: (2022)

Neuropathology and Therapeutic Intervention in Spinal and Bulbar Muscular Atrophy
por: Banno, Haruhiko, et al.
Publicado: (2009)

Current Status of Treatment of Spinal and Bulbar Muscular Atrophy
por: Tanaka, Fumiaki, et al.
Publicado: (2012)

Clinical Phenotyping and Biomarkers in Spinal and Bulbar Muscular Atrophy
por: Millere, Elina, et al.
Publicado: (2021)

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

Correlation of clinical and molecular features in spinal bulbar muscular atrophy
por: Fratta, Pietro, et al.
Publicado: (2014)

Cannot write session to /tmp/vufind_sessions/sess_lpjq1tk3r9qf28etv4ql95nm5f