Eficácia, segurança e aspectos regulatórios dos medicamentos órfãos para doenças raras: o caso Zolgensma® [PDF]
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 +3 more sources
International journal of health sciences, 2022 Disease and disorders are outlined as disablement of the normal state of living organism and annoyance of normal functioning of the body respectively. The ideal causes of the disorders are genetic factors, disease, stress or trauma. Genetic disorders are a pathological state provoked by one or more monstrosities in the genome.
M. Devi +3 more
openaire +2 more sources
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
core +7 more sources
Gene therapy of neurodegenerative diseases: achievements, developments, and clinical implementation challenges [PDF]
Биопрепараты: Профилактика, диагностика, лечение, 2023 Neurodegenerative diseases (NDDs) are promising objects for the development of gene therapy products, primarily, due to the possible cause of these diseases (disruption of a gene or several genes), lack of effective therapy, and negative impact on the ...
E. V. Melnikova +2 more
doaj +3 more sources
Perfil de notas técnicas para suporte técnico-científico à decisão judicial do medicamento Zolgensma® (onasemnogeno abeparvoveque) [PDF]
HU RevistaIntrodução: O aumento da judicialização da saúde tem destacado a importância dos centros de avaliação judicial em decisões baseadas em evidências.
Thisciane Ferreira Pinto Gomes +6 more
doaj +2 more sources
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
doaj +2 more sources
, 2022 Goed nieuws voor patiënten met de zeldzame erfelijke spierziekte spinale musculaire atrofie (SMA) en de zeldzame erfelijke stofwisselingsziekte metachromatische leukodystrofie (MLD).
Thielen, Frederick W.; id_orcid +4 more
core +10 more sources
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
doaj +2 more sources
Advanced therapy medicinal products (ATMPs) - An analysis of the global regulatory status. [PDF]
Br J Clin PharmacolAbstract Background A rapid progress in the development of advanced therapy medicinal products (ATMPs) has taken place over the past decades. The ATMPs hold great potential but face challenges in the clinical development and the regulatory process.
Cramer A +4 more
europepmc +2 more sources