Results 21 to 30 of about 160,551 (221)
Farmaci orfani e malattie rare: un confronto internazionale delle normative di riferimento
Farmeconomia: Health Economics and Therapeutic Pathways, 2001 Orphan drugs are defined as medicines with low economic returns, so that their production is not a profitable business far pharmaceutical companies.
S. Martina, C. Denti, L. Garattini
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Frontiers in Pharmacology, 2023 Introduction: In 2017 the drug chenodeoxycholic acid (CDCA) became unavailable to Dutch patients with the rare inborn error of metabolism cerebrotendinous xanthomatosis (CTX).
Natalja Bouwhuis +4 more
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Frontiers in Microbiology, 2022 Natural products from microorganisms are important sources for drug discovery. With the development of high-throughput sequencing technology and bioinformatics, a large amount of uncharacterized biosynthetic gene clusters (BGCs) in microorganisms have ...
Xingxing Li +10 more
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, 2021 Background:Several mitochondrial diseases are caused by pathogenic variants that impair membrane phospholipid remodeling. There are no US Food and Drug Administration (FDA)–approved therapies for these conditions.
M. Koenig +6 more
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Gut microbiota–derived short‐chain fatty acids regulate group 3 innate lymphoid cells in HCC
Hepatology, EarlyView., 2022 Abstract Background and Aims Type 3 innate lymphoid cells (ILC3s) are essential for host defense against infection and tissue homeostasis. However, their role in the development of HCC has not been adequately confirmed. In this study, we investigated the immunomodulatory role of short‐chain fatty acids (SCFAs) derived from intestinal microbiota in ILC3
Chupeng Hu +11 more
wiley +1 more source
Cryo‐EM structure of orphan G protein‐coupled receptor GPR21
MedComm, 2023 GPR21 belongs to class A orphan G protein‐coupled receptor (GPCR). The endogenous ligands for human GPR21 remain unidentified. GPR21 expression is associated with developing type 2 diabetes (T2DM), a multifactorial metabolic disease caused by pancreatic ...
Thian‐Sze Wong +8 more
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PharmacoEconomics - Open, 2022 The National Institute for Health and Care Excellence (NICE) is responsible for ensuring that patients in England and Wales can access clinically and cost-effective treatments. However, NICE's processes pose significant reimbursement challenges for treatments for rare diseases.
Dawn Lee +5 more
openaire +3 more sources
Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives (OD Expert Group) [PDF]
Frontiers in Pharmacology, 2021 Today policy makers face the challenge to devise a policy framework that improves orphan medicinal product (OMP) development by creating incentives to deliver treatments where there are none and to authorize innovative and transformative treatments where treatments already exist. The European Expert Group on Orphan Drug Incentives (hereafter, OD Expert
Annemieke Aartsma-Rus +2 more
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HYPERTENSION, 2020 No drug therapy has shown to limit abdominal aortic aneurysm (AAA) growth or rupture, and the understanding of the disease biology is incomplete; whereby, one challenge of vascular medicine is the development of good animal models and therapies for this ...
L. Cañes +7 more
semanticscholar +1 more source
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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