Results 1 to 10 of about 2,159 (173)

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   +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 Pharmacology
Onasemnogene 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, Yizhen Yin, Dan Yang, Mengyuan Liu, Caixia Ye, Ruiling Yan, Ruiman Li   +6 more
exaly   +6 more sources

Judicialization of Zolgensma in the Ministry of Health: costs and clinical profile of patients [PDF]

Revista de Saúde Pública
OBJECTIVE 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, Ellen Teixeira, Dayani Galato, Everton Nunes da Silva   +3 more
doaj   +5 more sources

Structural characterization of antibody-responses following Zolgensma treatment for AAV capsid engineering to expand patient cohorts [PDF]

Nature Communications
Monoclonal 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, Jane Hsi, Austin R. Nelson, Neeta Khandekar, Ann-Maree Huang, Nicholas JC Smith, Jon Zachary, Lindsay Potts, Michelle A. Farrar, Paul Chipman, Mohammad Ghanem, Ian E. Alexander, Grant J. Logan, Juha T. Huiskonen, Robert McKenna   +14 more
doaj   +6 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]

Pharmaceuticals
The recent introduction of the innovative therapy, onasemnogene abeparvovec (Zolgensma®), has revolutionized the spinal muscular atrophy (SMA) therapeutic landscape.
Rosanna Ruggiero, Nunzia Balzano, Maria Maddalena Nicoletti, Gabriella di Mauro, Federica Fraenza, Maria Rosaria Campitiello, Francesco Rossi, Annalisa Capuano   +7 more
doaj   +4 more sources

ID236 Zolgensma no tratamento da Atrofia Muscular Espinhal: Acordos de Acesso Gerenciado no mundo [PDF]

Jornal de Assistência Farmacêutica e Farmacoeconomia
Introdução 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, Amanda Oliveira Lyrio, Ana Carolina de Freitas Lopes, Luciene Fontes Schluckebier Bonan   +3 more
doaj   +4 more sources

Long-term comparative analysis of AAV9-mediated gene replacement therapies for spinal muscular atrophy in mice [PDF]

Nature Communications
Spinal 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, Qing Xie, Sarah J. Nath, Mojiao Tang, Hong Ma, Yasemin Özgür Günes, Tapan Sharma, Hao Liu, Mengtian Cui, Ailing Du, Mengjia Lu, Sophia Y. Liu, Boonying Wassamon, Mengyao Xu, Joseph Yunxi Wu, Qin Su, Timothy P. Fitzgibbons, Jinghua Liu, Fang Wan, Veena Kumanan, Ran He, Yijie Ma, Jun Yang, Heather L. Gray-Edwards, Thomas L. Gallagher, Phillip W. L. Tai, Guangping Gao, Jun Xie   +27 more
doaj   +3 more sources

New challenges in health technology assessment (HTA): the case of Zolgensma

Ciencia E Saude Coletiva, 2023
Abstract Rare or orphan diseases have played an important role in the pharmaceutical industry. On the other hand, the impact of new technologies derived from genomic research has been growing in this industry, with new drugs being launched on the market at unsustainable prices for health systems and patients.
Reinaldo Guimarães
exaly   +3 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
exaly   +3 more sources

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

Cureus, 2023
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 Administration (FDA) approved onasemnogene abeparvovec, SMN1 gene
Ogbonmide, Tolu, Rathore, Rajni, Rangrej, Shahid B, Hutchinson, Stedrea, Lewis, Marcia, Ojilere, Stephenie, Carvalho, Victoria, Kelly, Irenaissia   +7 more
exaly   +3 more sources