Results 21 to 30 of about 1,989 (180)
Acuerdo de riesgo compartido: si se pudo con Zolgensma, se puede con Risdiplam
Revista Derecho y SaludLa irrupción de terapias de alto costo, como las destinadas a la Atrofia Muscular Espinal (AME), ha exacerbado la tensión estructural entre el derecho al acceso a la salud y la sostenibilidad financiera del sistema sanitario argentino.
Sebastian Sandoval Junyent
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Alerta Farmacovigilancia Onasemnogén abeparvovec (▼Zolgensma): nuevas recomendaciones de control de la función hepática [PDF]
, 2023 En febrero de 2023, la Agencia Española de Medicamentos y Productos Sanitarios (AEMPS) emitió una alerta farmacológica acerca de casos de insuficiencia hepática aguda, incluyendo casos con desenlace mortal, en pacientes tratados con onasemnogén abeparvovec.
Inthamoussu, Maite, Galarraga, Florencia
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Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series [PDF]
BMC NeurologyBackground Spinal muscular atrophy (SMA) is caused by deletions or mutations in the survival of motor neuron (SMN) 1 gene resulting in progressive motor function loss, and additional disease-related complications, including dysphagia and respiratory ...
Melissa D. Svoboda +7 more
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PROPRIEDADE INTELECTUAL E O DIREITO À SAÚDE: O CASO DO USO DO ZOLGENSMA NO TRATAMENTO DA ATROFIA MUSCULAR ESPINHAL [PDF]
, 2023 Spinal Muscular Atrophy is a degenerative disease that causes the death of motor neurons, currently the drug treatment is still extremely expensive and difficult to access, among the treatment options, zolgensma stands out for being the only one ...
MARINHO, Matheus Maia
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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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, 2021 Tese de mestrado, Regulação e Avaliação do Medicamento e Produtos de Saúde, 2021, Universidade de Lisboa, Faculdade de FarmáciaA Atrofia Muscular Espinal (AME) é a principal causa genética de mortalidade infantil com uma incidência de aproximadamente 1 ...
Figueiredo, Ana Cristina Martins de
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Case report: A case of spinal muscular atrophy in a preterm infant: risks and benefits of treatment
Frontiers in Neurology, 2023 Spinal muscular atrophy (SMA) is a neuromuscular genetic disorder caused by the loss of lower motor neurons leading to progressive muscle weakness and atrophy.
Elisa Nigro +10 more
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Onasemnogene Abeparvovec (Zolgensma)
Canadian Journal of Health Technologies, 2021 In the March 2021, the CADTH Canadian Drug Expert Committee recommended that onasemnogene abeparvovec be reimbursed for the treatment of pediatric patients with 5q spinal muscular atrophy (SMA) with biallelic mutations in the survival motor neuron 1 (SMN1) gene, if certain conditions were met.
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Newer advances in the treatment of Duchenne muscular dystrophy and spinal muscular atrophy
Journal of Current Research in Scientific Medicine, 2019 Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) are two common and important Inherited neuromuscular disorders which have witnessed immense advances in their treatment owing to ongoing developments in gene therapy.
Mukesh Kumar, Venugopalan Y Vishnu
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