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
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
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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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Current State of Human Gene Therapy: Approved Products and Vectors
Pharmaceuticals, 2023 In the realm of gene therapy, a pivotal moment arrived with Paul Berg’s groundbreaking identification of the first recombinant DNA in 1972. This achievement set the stage for future breakthroughs.
Aladdin Y. Shchaslyvyi +3 more
doaj +1 more source
A versatile toolkit for overcoming AAV immunity
Frontiers in Immunology, 2022 Recombinant adeno-associated virus (AAV) is a promising delivery vehicle for in vivo gene therapy and has been widely used in >200 clinical trials globally.
Xuefeng Li +5 more
doaj +1 more source
Immunomodulation in Administration of rAAV: Preclinical and Clinical Adjuvant Pharmacotherapies
Frontiers in Immunology, 2021 Recombinant adeno-associated virus (rAAV) has attracted a significant research focus for delivering genetic therapies to target cells. This non-enveloped virus has been trialed in many clinical-stage therapeutic strategies but important obstacle in ...
Wing Sum Chu, Joanne Ng
doaj +1 more source
Zolgensma-hoito (onasemnogeeniabeparvoveekki) SMA-taudin hoidossa : Uusien sairaalalääkkeiden arviointi [PDF]
, 2020 Spinaalinen lihasatrofia (SMA) on harvinainen perinnöllinen neuromuskulaarinen sairaus. SMA-tauti johtaa lihasten rappeutumiseen ja lihasheikkouteen, mikä estää motoristen toimintojen kehittymisen ja ylläpitämisen.
Hyvärinen, Antti +2 more
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