Results 141 to 150 of about 11,812,623 (407)

Experimental designs for small randomised clinical trials: An algorithm for choice [PDF]

, 2013
Background: Small clinical trials are necessary when there are difficulties in recruiting enough patients for conventional frequentist statistical analyses to provide an appropriate answer.
Alberti, Corinne, Bajard, Agathe, Ballot, Clément, Castellan, Anne Charlotte, Caudri, Daan, Chabaud, Sylvie, Chiron, Catherine, Cornu, Catherine, Fisch, Roland, Guerrini, Renzo, Kassai, Behrouz, Kurbatova, Polina, Nony, Patrice, Ogungbenro, Kayode, Pons, Gerard, Rosati, Anna, Tiddens, Harm   +16 more
core   +5 more sources

Inhibition of TGF‐β Increases Bone Volume and Strength in a Mouse Model of Osteogenesis Imperfecta

JBMR Plus, 2021
Osteogenesis imperfecta (OI), is a genetic disorder of bone fragility caused by mutations in collagen I or proteins involved in collagen processing. Previous studies in mice and human OI bones have shown that excessive activation of TGF‐β signaling plays
Benjamin Greene, Ryan J Russo, Shannon Dwyer, Katie Malley, Errin Roberts, Joseph Serrielo, Peter Piepenhagen, Sheila Cummings, Susan Ryan, Christine Zarazinski, Sasidhar Uppuganti, Nikolai Bukanov, Jeffry S Nyman, Megan K Cox, Shiguang Liu, Oxana Ibraghimov‐Beskrovnaya, Yves Sabbagh   +16 more
doaj   +1 more source

To what degree are orphan drugs patient-centered? A review of the current state of clinical research in rare diseases

Orphanet Journal of Rare Diseases, 2020
Background Over the past 30 years, the healthcare industry has increasingly turned its attention to rare diseases. Regulators have emphasized the need for clinical research in this area to be patient-centered.
S. Lanar, C. Acquadro, James Seaton, I. Savre, B. Arnould   +4 more
semanticscholar   +1 more source

Rare ophthalmology diseases

romanian journal of ophthalmology, 2019
Rare ocular pathology has an important impact on the quality of life of patients because often the damage is bilateral and, although asymmetric, causes a significant decrease in visual acuity. Because it may be asymptomatic until a relatively late stage, diagnosis is frequently delayed. A general understanding of the disease pathophysiology, diagnosis,
Sanziana Istrate, Aida Geamanu, Cristina Alexandrescu, Raluca Iancu, Elena Angelica Sburlan, Mihai Ghita, Emil Ungureanu, Liliana-Mary Voinea, Ruxandra Pirvulescu, Ciuluvica Radu   +9 more
openaire   +3 more sources

Evaluation of KRAS and NRAS mutations in metastatic colorectal cancer: an 8‐year study of 10 754 patients in Turkey

Molecular Oncology, EarlyView.
This nationwide study evaluated KRAS and NRAS mutations in 10 754 Turkish patients with metastatic colorectal cancer. The results revealed a mutation frequency of 51.1%, with 46.6% having KRAS mutations, 4.5% having NRAS mutations, and 48.5% being wild‐type for both.
Gozde Kavgaci, Izzet Akiva, Yavuz Hakan Ozon, Hakan Berkil, Huseyin Karadayi, Omer Dizdar, Suayib Yalcin   +6 more
wiley   +1 more source

Modelling the Neuroanatomical Progression of Alzheimer's Disease and Posterior Cortical Atrophy [PDF]

arXiv, 2020
In order to find effective treatments for Alzheimer's disease (AD), we need to identify subjects at risk of AD as early as possible. To this end, recently developed disease progression models can be used to perform early diagnosis, as well as predict the subjects' disease stages and future evolution.
arxiv  

Circulating tumor DNA monitoring and blood tumor mutational burden in patients with metastatic solid tumors treated with atezolizumab

Molecular Oncology, EarlyView.
In patients treated with atezolizumab as a part of the MyPathway (NCT02091141) trial, pre‐treatment ctDNA tumor fraction at high levels was associated with poor outcomes (radiographic response, progression‐free survival, and overall survival) but better sensitivity for blood tumor mutational burden (bTMB).
Charles Swanton, Russell W. Madison, Candice Francheska B. Tambaoan, Funda Meric‐Bernstam, Christopher J. Sweeney, Razelle Kurzrock, Howard A. Burris III, David R. Spigel, Hanna Tukachinsky, Jason Hughes, Julia Malato, Bongin Yoo, Tania Szado, Cheryl Schwab, Lincoln W. Pasquina, Amaya Gasco, Katja Schulze, Claire F. Friedman   +17 more
wiley   +1 more source

An integrative knowledge graph for rare diseases, derived from the Genetic and Rare Diseases Information Center (GARD)

Journal of Biomedical Semantics, 2020
The Genetic and Rare Diseases (GARD) Information Center was established by the National Institutes of Health (NIH) to provide freely accessible consumer health information on over 6500 genetic and rare diseases. As the cumulative scientific understanding
Qian Zhu, Dac-Trung Nguyen, Ivan Grishagin, Noel Southall, E. Sid, Anne R. Pariser   +5 more
semanticscholar   +1 more source

Strategies to reduce the cancer burden and improve access to effective and affordable cancer interventions in Europe

Molecular Oncology, EarlyView.
Comprehensive cancer centre (CCCs) and CCCs of Excellence (CCCoE) integration in healthcare. Through outreach to surrounding community hospitals, CCCs enable wider access to top‐clinical cancer treatments and care, thereby facilitating the swift enrolment of patients into data‐rich clinical trials (PI‐initiated trials testing new concepts, drug ...
Anton Berns, Ulrik Ringborg, Julio Celis, Bengt Jönsson, Michael Baumann   +4 more
wiley   +1 more source

Drugs for exceptionally rare diseases: a commentary on Hughes et al [PDF]

, 2006
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., McCabe, C., Raftery, J., Tsuchiya, A.   +3 more
core