Quantitative retrospective natural history modeling for orphan drug development
نویسندگان
چکیده
منابع مشابه
Orphan drug: Development trends and strategies
The growth of pharma industries has slowed in recent years because of various reasons such as patent expiries, generic competition, drying pipelines, and increasingly stringent regulatory guidelines. Many blockbuster drugs will loose their exclusivity in next 5 years. Therefore, the current economic situation plus the huge generic competition shifted the focus of pharmaceutical companies from t...
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BACKGROUND Lysosomal storage disorders are a heterogeneous group of approximately 50 monogenically inherited orphan conditions. A defect leads to the storage of complex molecules in the lysosome, and patients develop a complex multisystemic phenotype of high morbidity often associated with premature death. More than 30 years ago the Orphan Drug Act of 1983 passed the United States legislation i...
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As research into drugs targeting rare or ‘‘orphan’’ diseases has stepped up in recent years, so too has the need for a robust business model capable of bringing promising orphan drugs into the marketplace. The earliest step took place in 1983, when President Ronald Reagan signed into law the Orphan Drug Act. More than a quarter-century later, the orphan disease community is looking again to Was...
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The need and opportunity to discover therapeutics for rare or orphan diseases are enormous. Due to limited prevalence and/or commercial potential, of the approximately 6000 orphan diseases (defined by the FDA Orphan Drug Act as <200 000 US prevalence), only a small fraction (5%) is of interest to the biopharmaceutical industry. The fact that drug development is complicated, time-consuming and e...
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The ubiquitin-proteasome system (UPS) plays a major role in selective protein degradation and regulates various cellular events. Approval of bortezomib for the treatment of multiple myeloma validated the proteasome as an anticancer target. In order to find drug candidates targeting the ubiquitin-dependent protein degradation, we paid an attention to inhibitors against three enzymes, ubiquitin-a...
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ژورنال
عنوان ژورنال: Journal of Inherited Metabolic Disease
سال: 2020
ISSN: 0141-8955,1573-2665
DOI: 10.1002/jimd.12304