Comprehensive analysis of adverse events associated with onasemnogene abeparvovec (Zolgensma) in spinal muscular atrophy patients: insights from FAERS database [PDF]
Frontiers in PharmacologyOnasemnogene Abeparvovec (Zolgensma) is a gene therapy for the treatment of Spinal Muscular Atrophy (SMA) with improved motor neuron function and the potential for a singular treatment.
Wenwen Zhang +6 more
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Pricing Zolgensma – the world’s most expensive drug [PDF]
Journal of Market Access & Health Policy, 2022 A heated discussion has recently broken out in Europe about the price of Zolgensma, ‘the most expensive drug ever’ [...]
Mark Nuijten
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Judicialization of Zolgensma in the Ministry of Health: costs and clinical profile of patients [PDF]
Revista de Saúde PúblicaOBJECTIVE To investigate the costs and profile of patients who have filed a lawsuit against the Ministry of Health for the treatment of spinal muscular atrophy (SMA) with the onasemnogene abeparvovec (Zolgensma®).
Ana Katheryne Miranda Kretzschmar +3 more
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Structural characterization of antibody-responses following Zolgensma treatment for AAV capsid engineering to expand patient cohorts [PDF]
Nature CommunicationsMonoclonal antibodies are useful tools to dissect the neutralizing antibody response against the adeno-associated virus (AAV) capsids that are used as gene therapy delivery vectors.
Mario Mietzsch +14 more
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Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System [PDF]
PharmaceuticalsThe recent introduction of the innovative therapy, onasemnogene abeparvovec (Zolgensma®), has revolutionized the spinal muscular atrophy (SMA) therapeutic landscape.
Rosanna Ruggiero +7 more
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Experiences and the psychosocial situation of parental caregivers of children with spinal muscular atrophy against the background of new treatment options: a qualitative interview study [PDF]
BMC PsychologyBackground Spinal muscular atrophy is a rare neurodegenerative disorder in children which leads untreated to muscle wasting, respiratory impairments, and a shortened life expectancy.
Maja Brandt +5 more
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Long-term comparative analysis of AAV9-mediated gene replacement therapies for spinal muscular atrophy in mice [PDF]
Nature CommunicationsSpinal muscular atrophy (SMA) results from a deficiency of the survival motor neuron (SMN) protein. Zolgensma, an adeno-associated virus (AAV)-based SMN1 gene-replacement therapy, is approved for SMA, though its long-term efficacy and safety remain ...
Xiupeng Chen +27 more
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Crowd Funding for Orphan Drugs: The Case of Baby Pia
Frontiers in Pharmacology, 2021 Medical crowdfunding is a relatively new strategy to obtain access to orphan drugs. The case of Baby Pia, a Belgian girl with SMA type 1 for whom in 2018 more than $ 2.1 million was raised to get her treated with Zolgensma®, illustrates well the ...
Wim Pinxten
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The role of vascular pathology in SMA: implications for pathogenesis and treatment [PDF]
Frontiers in Cellular NeuroscienceSpinal muscular atrophy (SMA) has become a paradigm for precision medicine following the development of transformative therapies targeting the disease-causing deficiency of survival motor neuron (SMN) protein.
Simon H. Parson, Hazel Allardyce
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Gene Therapy Medicinal Products: Non-clinical Safety Studies
Безопасность и риск фармакотерапии, 2023 Currently, gene therapy medicinal products (GTMPs) are actively developed in many countries, including the Russian Federation. However, the use of GTMPs raises class-specific safety concerns.The aim of the study was to determine the main requirements for
O. V. Astapova, A. A. Berchatova
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