Results 21 to 30 of about 2,159 (173)
The Multiple Criteria Qualitative Value-Based Pricing Framework "MARIE" for Novel Cell and Gene Therapy. [PDF]
Clin Pharmacol TherWhen setting the price of cell and gene therapies in Japan, the factors considered include the manufacturing and operating costs of the new drug or a comparator, leading to discrepancies between price and values, in particular those specific to cell and gene therapies.
Takami A, Igarashi A.
europepmc +2 more sources
, 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
core +5 more sources
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
core +1 more source
Impact of Intermittent Positive Pressure Breathing on Thoracic Growth in Children With Type 1 Spinal Muscular Atrophy. [PDF]
Pediatr PulmonolABSTRACT Introduction The emergence of new disease‐modifying treatments for type I spinal muscular atrophy (SMA I) has led to a paradigm shift in the respiratory management of these patients. Accompanying pulmonary and thoracic growth appears to be a key factor in their morbidity and mortality. The role of Intermittent Positive Pressure Breathing (IPPB)
Thébault C +9 more
europepmc +2 more sources
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
doaj +1 more source
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
doaj +1 more source
New prospects for the treatment of Spinal Muscular Atrophy
Journal of Education, Health and Sport, 2019 Introduction: Spinal muscular atrophy (SMA) is one of the most common genetically determined causes of infant and young child death. The aim of the study: Review of medical literature on therapeutic strategies used in the treatment of SMA. Material and
Julita Poleszak +6 more
doaj +3 more sources
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.
openaire +2 more sources
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
doaj +1 more source
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
doaj +1 more source