Cargando…

Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma)

Spinal Muscular Atrophy (SMA) is a genetic disease that causes weakness and wasting in the voluntary muscles of infants and children. SMA has been the leading inherited cause of infant death. More specifically, SMA is caused by the absence of the SMN1 gene. In May 2019, the Food and Drug Administrat...

Descripción completa

Detalles Bibliográficos
Autores principales:	Ogbonmide, Tolu, Rathore, Rajni, Rangrej, Shahid B, Hutchinson, Stedrea, Lewis, Marcia, Ojilere, Stephenie, Carvalho, Victoria, Kelly, Irenaissia
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Cureus 2023
Materias:	Genetics
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10104684/ https://www.ncbi.nlm.nih.gov/pubmed/37065340 http://dx.doi.org/10.7759/cureus.36197

Ejemplares similares

Onasemnogene abeparvovec for spinal muscular atrophy
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 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)

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)

Combination Therapy with Nusinersen and Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy Type I
por: Mirea, Andrada, 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)

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

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)

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)

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)

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

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)

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

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)

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)

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)

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

Pricing Zolgensma – the world’s most expensive drug
por: Nuijten, Mark
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)

Spinal Muscular Atrophy (SMA) Subtype Concordance in Siblings: Findings From the Cure SMA Cohort
por: Jones, Cynthia C., et al.
Publicado: (2020)

Eighteen Years with Spinal Muscular Atrophy (SMA) Type 1
por: Valencia, Borja, et al.
Publicado: (2013)

Burden of Spinal Muscular Atrophy (SMA) on Patients and Caregivers in Canada
por: McMillan, H.J., et al.
Publicado: (2021)

SMA-MAP: A Plasma Protein Panel for Spinal Muscular Atrophy
por: Kobayashi, Dione T., et al.
Publicado: (2013)

Measuring Fatigue and Fatigability in Spinal Muscular Atrophy (SMA): Challenges and Opportunities
por: Rodriguez-Torres, Rafael S., et al.
Publicado: (2023)

The Benefits of Integrative Medicine for Pain Management in Oncology: A Narrative Review of the Current Evidence
por: Carvalho, Victoria, et al.
Publicado: (2023)

A call to introduce newborn screening for spinal muscular atrophy (SMA) in Scotland
por: Gillingwater, Thomas H., et al.
Publicado: (2022)

Systematic Literature Review to Assess Economic Evaluations in Spinal Muscular Atrophy (SMA)
por: Paracha, Noman, et al.
Publicado: (2021)

Alambres musculares (SMA) /
por: Hernández Ramírez, Edgar
Publicado: (2001)

Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)
por: Zanetta, Chiara, et al.
Publicado: (2014)

Spontaneous Breathing Pattern as Respiratory Functional Outcome in Children with Spinal Muscular Atrophy (SMA)
por: LoMauro, A., et al.
Publicado: (2016)

Cannot write session to /tmp/vufind_sessions/sess_lj85jjcj6ua56gu7r7bj3d9na3