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
exaly +4 more sources
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
doaj +5 more sources
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.
Yizhen Yin, Ruiman Li
exaly +4 more sources
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
doaj +5 more sources
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
doaj +2 more sources
Novos desafios na avaliação de tecnologias em saúde (ATS): o caso Zolgensma
Ciencia E Saude Coletiva, 2023 Resumo As doenças raras ou órfãs entraram de modo importante nos negócios da indústria farmacêutica. Por outro lado, o impacto das novas tecnologias derivadas da pesquisa genômica vêm tendo presença crescente nessa indústria, com novos medicamentos sendo lançados no mercado a preços não sustentáveis para sistemas de saúde e pacientes.
Reinaldo Guimarães +1 more
exaly +3 more sources
ID236 Zolgensma no tratamento da Atrofia Muscular Espinhal: Acordos de Acesso Gerenciado no mundo
Jornal de Assistência Farmacêutica e FarmacoeconomiaIntrodução Os Acordos de Acesso Gerenciado (ACR) são estratégias para mitigar incertezas relacionadas às tecnologias de alto custo, como o Zolgensma© (onasemnogeno abeparvoveque), medicamento indicado para o tratamento da Atrofia Muscular Espinhal ...
Laís Lessa Neiva Pantuzza +3 more
doaj +3 more sources
Cadernos Ibero-Americanos de Direito Sanitário, 2019 Objetivo: discutir o processo regulatório de medicamentos para doenças raras no Brasil, com base no caso Zolgensma®, e avaliar criticamente as evidências disponíveis até o momento sobre a eficácia e a segurança do Zolgensma® no tratamento da atrofia ...
Rachel Riera +2 more
doaj +4 more sources
Cytotherapy, 2022 Drug prices are regarded as one of the most influential factors in determining accessibility and affordability to novel therapies. Cell and gene therapies such as OTL-200 (brand name: Libmeldy) and AVXS-101 (brand name: Zolgensma) with (expected) list prices of 3.0 million EUR and 1.9 million EUR per treatment, respectively, spark a global debate on ...
Frederick W Thielen +2 more
exaly +3 more sources
Journal of Market Access & Health Policy, 2021 Background: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results.
Douglas M Sproule +2 more
exaly +2 more sources