Economic Evaluation in Duchenne Muscular Dystrophy: Model Frameworks for Cost-Effectiveness Analysis
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چکیده
منابع مشابه
Economic Evaluation in Duchenne Muscular Dystrophy: Model Frameworks for Cost-Effectiveness Analysis
BACKGROUND Several treatments are on the horizon for Duchenne muscular dystrophy (DMD), a terminal orphan disease. In many jurisdictions, decisions regarding pricing and reimbursement of these health technologies comprise evidence of value for money. OBJECTIVE The objective of this study was to develop a cost-effectiveness model based on the Duchenne muscular dystrophy Functional Ability Self...
متن کاملP164: Adeno-Associated Viral Vectors in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (BMD) is an inherited X-link disease. The incidence of this muscle-wasting disease is 1:5000 male live births. Mutation in the gene coding for dystrophin is the main cause of BMD. Most cases of this disease succumb to respiratory and cardiac failure in 3rd to 4th decades. The slow progression of BMD and recent achievement of gene therapies make it as an appropriate c...
متن کاملFunctional Evaluation for Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a genetic, progressive and irreversible disorder linked to the X chromosome, which affects one in every 3,500 male children born alive. Induces muscle weakness as result of a deficiency or absence of the protein dystrophin, resulting in functional motor abilities loss, which starts when the child is between three and seven years old. Falls and difficulty run...
متن کاملDuchenne muscular dystrophy An overview of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) affects approximately 1 in 3,500 live male births [1]. It is caused by a large variety of mutations in the dystrophin gene. Because of these mutations, the body can no longer make dystrophin which is a protein important for stabilisation of the muscle cell during a contraction. Without dystrophin, muscle cells are damaged and slowly replaced by fat and scar tis...
متن کاملMolecular deletion analysis in Duchenne muscular dystrophy.
Study of 165 unrelated patients with X linked muscular dystrophy (117 with Duchenne and 48 with Becker dystrophy) has shown nine Duchenne cases (8% of the total) where a molecular deletion was detected using probes pERT87 or XJ1.1. No cytogenetic abnormalities were detectable in this unselected series of patients and no clear clinical or other differences were found between deletion and non-del...
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ژورنال
عنوان ژورنال: PharmacoEconomics
سال: 2016
ISSN: 1170-7690,1179-2027
DOI: 10.1007/s40273-016-0461-5