Results 11 to 20 of about 160,551 (221)
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
The Orphan Drug Act and the Development of Stem Cell-Based Products for Rare Diseases [PDF]
Cell Stem Cell, 2010 The Orphan Drug Act encourages the development of products for rare diseases and conditions. Many conditions that stand to benefit from stem cell-based products are rare diseases. We address the Orphan Drug Act in relation to the development of stem cell-based products.
Scott N. Freeman +3 more
openaire +3 more sources
Regulatory Approach To Orphan Medicines In The United States And EU
Varna Medical Forum, 2021 Introduction : Rare diseases represent a growing significant public health problem and a challenge for health systems. For most diseases no treatment has been developed yet.
A. Serbezova +2 more
semanticscholar +1 more source
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
Frontiers in Immunology, 2021 Small heterodimer partner interacting leucine zipper protein (SMILE) is an orphan nuclear receptor and a member of the bZIP family of proteins. We investigated the mechanism by which SMILE suppressed the development of inflammatory bowel disease (IBD ...
SeungCheon Yang +16 more
doaj +1 more source
Small heterodimer partner-targeting therapy inhibits systemic inflammatory responses through mitochondrial uncoupling protein 2. [PDF]
PLoS ONE, 2013 The orphan nuclear receptor, small heterodimer partner (SHP), appears to play a negative regulatory role in innate immune signaling. Emerging evidence warrants further study on the therapeutic targeting of SHP to suppress excessive and deleterious ...
Chul-Su Yang +8 more
doaj +1 more source
Gene and Cell Therapies Overview Under the Light of Health Economics [PDF]
Health Economics and Management Review, 2022 With the increase in drug development studies for rare diseases, gene therapies have recently come to the fore more frequently. In addition to orphan drugs used in the treatment of rare diseases, advanced therapy medicinal products have been developed ...
Ekin Begum Karahan, Guvenc Kockaya
doaj +1 more source
Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders [PDF]
Orphanet Journal of Rare Diseases, 2011 Orphan drugs are often approved under exceptional circumstances, requiring submission of additional data on safety and effectiveness through registries. These registries are mainly focused on one drug only and data is frequently incomplete. Some registries also address phenotypic heterogeneity and natural history data and publications on these aspects ...
Hollak, C.E.M. +3 more
openaire +8 more sources
Is the Orphan Drug Industry the Proper Scapegoat for Unethical Research Funding?
Voices in Bioethics, 2019 The mainstream media and general public have expressed frustration in recent months with the confluence of capitalism and the pharmaceutical research and development enterprise.
Michael Menconi
doaj +1 more source
Pharmaceuticals, 2023 The iron chelating orphan drug deferiprone (L1), discovered over 40 years ago, has been used daily by patients across the world at high doses (75–100 mg/kg) for more than 30 years with no serious toxicity.
George J. Kontoghiorghes
doaj +1 more source