Results 81 to 90 of about 88,754 (289)
In silico and in vitro drug screening identifies new therapeutic approaches for Ewing sarcoma. [PDF]
, 2016 The long-term overall survival of Ewing sarcoma (EWS) patients remains poor; less than 30% of patients with metastatic or recurrent disease survive despite aggressive combinations of chemotherapy, radiation and surgery.
Alturkmani, Hani +11 more
core +3 more sources
Precision Medicines’ Impact on Orphan Drug Designation
Clinical and Translational Science, 2019 The incentives provided under the Orphan Drug Act (ODA) have been credited for catalyzing the marketing approval of drugs for the treatment of rare diseases by the US Food and Drug Administration.
Christine M. Mueller +2 more
doaj +1 more source
Orphanet Journal of Rare Diseases, 2020 Background Pricing and reimbursement decisions for orphan drugs are faced with differences access between European countries depending on each reimbursement policies, evaluation processes and timings.
Xavier Badia +5 more
doaj +1 more source
Advanced Intelligent Discovery, EarlyView.Predictive models successfully screen nanoparticles for toxicity and cellular uptake. Yet, complex biological dynamics and sparse, nonstandardized data limit their accuracy. The field urgently needs integrated artificial intelligence/machine learning, systems biology, and open‐access data protocols to bridge the gap between materials science and safe ...
Mariya L. Ivanova +4 more
wiley +1 more source
Drugs for exceptionally rare diseases: a commentary on Hughes et al [PDF]
Recently in this journal, Hughes and colleagues discussed special funding status to ultra-orphan drugs. They concluded that there should be a uniform policy for the provision of orphan drugs across Europe; that complete restriction was impractical, and ...
Claxton, K. +3 more
core
Making rareness count: testing and pricing orphan drugs [PDF]
, 2016 This paper examines the testing and the pricing of orphan drugs, e.g. drugs for patients suffering from rare diseases. Due to the small size of these populations, orphan molecules question established evidentiary practices, namely randomized controlled ...
Doganova, Liliana, Rabeharisoa, Vololona
core +3 more sources
Ethical hurdles in the prioritization of oncology care [PDF]
, 2016 With finite resources, healthcare payers must make difficult choices regarding spending and the ethical distribution of funds. Here, we describe some of the ethical issues surrounding inequity in healthcare in nine major European countries, using cancer ...
Capri, Stefano +12 more
core +1 more source
American Journal of Medical Genetics Part B: Neuropsychiatric Genetics, EarlyView.ABSTRACT We examined the relationship between adverse childhood experiences (ACEs) and epigenetic age acceleration (EAA) in adulthood as measured by second and third generation epigenetic clocks by performing a systematic review of the literature. The electronic databases MEDLINE and EMBASE were searched on 17 July 2023.
Matthew Green +2 more
wiley +1 more source
Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries
Frontiers in Pharmacology, 2019 Objective: To review the reimbursement recommendations issued by selected European health technology assessment agencies for orphan drugs and the reimbursement status of these drugs; to assess the relationship between the type of recommendation and ...
Ewa Stawowczyk +16 more
doaj +1 more source
American Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.ABSTRACT Myhre syndrome is an ultrarare genetic disease characterized by short stature, distinct craniofacial features, cardiovascular and respiratory fibrosis and stenosis, neurodevelopmental delays, autism, intellectual disability, and hearing loss. The natural history of Myhre syndrome is still not fully understood due to a small patient population ...
Mary K. Young +6 more
wiley +1 more source