The orphan drug dichloroacetate reduces amyloid beta-peptide production whilst promoting non-amyloidogenic proteolysis of the amyloid precursor protein. [PDF]
PLoS ONE, 2022 The amyloid cascade hypothesis proposes that excessive accumulation of amyloid beta-peptides is the initiating event in Alzheimer's disease. These neurotoxic peptides are generated from the amyloid precursor protein via sequential cleavage by β- and γ ...
Edward T Parkin +3 more
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Predictive Analysis for First Submission of Generic Drug Application for Orphan Drug Products Using Random Survival Forest. [PDF]
Clin Transl SciABSTRACT Rare diseases affect a small population of patients, resulting in low incentives for developing orphan drug products (ODPs). The United States Congress passed the Orphan Drug Act of 1983 to incentivize pharmaceutical manufacturers to develop drugs to treat rare diseases.
Hopefl R +6 more
europepmc +2 more sources
Actas Dermo-Sifiliográficas, 2022 Resumen: Introducción: El abordaje terapéutico de las manifestaciones cutáneas de las enfermedades raras es complejo. El objetivo principal de este trabajo consistió en determinar el impacto de la formulación magistral de dispensación hospitalaria en la
M. Oro-Ayude +4 more
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Experiencia clínica con medicamentos huérfanos para enfermedades raras metabólicas
Anales de Pediatría, 2022 Resumen: Introducción: Los errores congénitos del metabolismo son un grupo muy heterogéneo de enfermedades raras. La mayoría se pueden tratar con dieta y sustitución enzimática.Existen pocos pacientes y pocos estudios publicados en estas enfermedades ...
Alicia Caso-González +6 more
doaj +1 more source
Orphan drug development in alpha-1 antitypsin deficiency
Scientific Reports, 2022 Alpha-1 antitrypsin deficiency (AATD, OMIM #613490) is a rare metabolic disorder affecting lungs and liver. The purpose of this study is to assess the impact of the US orphan drug act on AATD by providing a quantitative clinical-regulatory insight into ...
Franziska C. Trudzinski +4 more
doaj +1 more source
The drug development pipeline for glioblastoma—A cross sectional assessment of the FDA Orphan Drug Product designation database [PDF]
PLOS ONE, 2021 Background Glioblastoma (GBM) is the most common malignant brain tumour among adult patients and represents an almost universally fatal disease. Novel therapies for GBM are being developed under the orphan drug legislation and the knowledge on the molecular makeup of this disease has been increasing rapidly ...
Pascal Johann, Dominic Lenz, Markus Ries
openaire +4 more sources
Changing incentives to ACCELERATE drug development for paediatric cancer
Cancer Medicine, 2023 Background More effective incentives are needed to motivate paediatric oncology drug development, uncoupling it from dependency on adult drug development.
Teresa deRojas +10 more
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The case of eculizumab: litigation and purchases by the Brazilian Ministry of Health
Revista de Saúde Pública, 2020 OBJECTIVES This study examined the purchases of eculizumab, a high-cost monoclonal antibody used in the treatment of rare diseases by Brazilian federal agencies, in terms of purchased quantities, expenditures, and prices.
Rosângela Caetano +4 more
doaj +2 more sources
New role of extemporaneous manufacturing in regulating drug products access onto the market
Фармация и фармакология (Пятигорск), 2023 The aim of the work was to study the legal aspects of the legislative regulation for manufacturing medicines in a pharmacy organization.Materials and methods.
A. V. Alekhin +5 more
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One‐pot synthesis of 6‐aminohexanoic acid from cyclohexane using mixed‐species cultures
Microbial Biotechnology, 2021 Summary 6‐Aminohexanoic acid (6AHA) is a vital polymer building block for Nylon 6 production and an FDA‐approved orphan drug. However, its production from cyclohexane is associated with several challenges, including low conversion and yield, and severe ...
Lisa Bretschneider +4 more
doaj +1 more source