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. Manufacturer prescribing information, article bibliographies, and data from ClinicalTrials.gov were incorporated in the reviewed data. STUDY SELECTION/DATA EXTRACTION: All studies registered on ClinicalTrials.gov were incorporated in the reviewed data. DATA SYNTHESIS: Onasemnogene is the first agent for SMA utilizing gene therapy to directly provide survival motor neuron 1 (SMN1) gene to produce SMN protein. Four publications of 1 clinical trial, 1 comparison study of treatment effects, and 1 combination therapy case series have been published. RELEVANCE TO PATIENT CARE AND CLINICAL PRACTICE: Onasemnogene is a one time dose approved by the Food and Drug Administration for SMA patients <2 years old who possess mutations in both copies of the SMN1 gene. CONCLUSION: Onasemnogene appears to be an efficacious therapy for younger pediatric patients with SMA type 1. Concerns include drug cost and potential liver toxicity. Long-term benefits and risks have not been determined.
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. Manufacturer prescribing information, article bibliographies, and data from ClinicalTrials.gov were incorporated in the reviewed data. STUDY SELECTION/DATA EXTRACTION: All studies registered on ClinicalTrials.gov were incorporated in the reviewed data. DATA SYNTHESIS: Onasemnogene is the first agent for SMA utilizing gene therapy to directly provide survival motor neuron 1 (SMN1) gene to produce SMN protein. Four publications of 1 clinical trial, 1 comparison study of treatment effects, and 1 combination therapy case series have been published. RELEVANCE TO PATIENT CARE AND CLINICAL PRACTICE: Onasemnogene is a one time dose approved by the Food and Drug Administration for SMApatients <2 years old who possess mutations in both copies of the SMN1 gene. CONCLUSION: Onasemnogene appears to be an efficacious therapy for younger pediatric patients with SMA type 1. Concerns include drug cost and potential liver toxicity. Long-term benefits and risks have not been determined.
Authors: Siiri Sarv; Tiina Kahre; Eve Vaidla; Sander Pajusalu; Kai Muru; Haide Põder; Katrin Gross-Paju; Sandra Ütt; Riina Žordania; Inga Talvik; Eve Õiglane-Shlik; Kristina Muhu; Katrin Õunap Journal: Front Genet Date: 2021-12-22 Impact factor: 4.599
Authors: John N Milligan; Jessica L Larson; Stela Filipovic-Sadic; Walairat Laosinchai-Wolf; Ya-Wen Huang; Tsang-Ming Ko; Kristin M Abbott; Henny H Lemmink; Minna Toivonen; Johanna Schleutker; Caren Gentile; Vivianna M Van Deerlin; Huiping Zhu; Gary J Latham Journal: J Mol Diagn Date: 2021-03-30 Impact factor: 5.341
Authors: Katharina E Meijboom; Viola Volpato; Jimena Monzón-Sandoval; Joseph M Hoolachan; Suzan M Hammond; Frank Abendroth; Olivier G de Jong; Gareth Hazell; Nina Ahlskog; Matthew Ja Wood; Caleb Webber; Melissa Bowerman Journal: JCI Insight Date: 2021-07-08