Results 161 to 170 of about 69,645 (303)

Isolation and Properties of the Abnormal Factor IX Molecule of Hemophilia BM [PDF]

open access: bronze, 1977
Bjarne Østerud   +3 more
openalex   +1 more source

Halfspace Depth

open access: yesWIREs Computational Statistics, Volume 17, Issue 3, September 2025.
The halfspace depth generalizes quantiles to multivariate data. This is a bagplot—a depth‐based analog of a boxplot. It succinctly captures the geometry of the bivariate dataset (blue/red points) and identifies the four red points in the top left corner as deviating from the general pattern of the data.
Stanislav Nagy
wiley   +1 more source

Recent Advances in mRNA Delivery Systems for Cancer Therapy

open access: yesAdvanced Science, Volume 12, Issue 29, August 7, 2025.
This review systematically investigates the applications of mRNA therapy in cancer treatment, with particular emphasis on nonviral delivery systems, targeting strategies, stimulus‐responsive systems, and local delivery methods. Concluding with a meticulous evaluation, the review sheds light on the prevailing challenges while illuminating promising ...
Zheng Zhang   +9 more
wiley   +1 more source

Impact of Model‐Informed Drug Development on Drug Development Cycle Times and Clinical Trial Cost

open access: yesClinical Pharmacology &Therapeutics, Volume 118, Issue 2, Page 378-385, August 2025.
Model‐informed drug development (MIDD) integrates data to quantify benefit/risk informing objective drug discovery and development decisions. An additional critical benefit of MIDD is postulated to be improvement in trial and program efficiencies. While the application of MIDD has grown, there have been no clear examples across programs to demonstrate ...
Vaishali Sahasrabudhe   +4 more
wiley   +1 more source

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

open access: yesNew England Journal of Medicine, 2017
S. Rangarajan   +10 more
semanticscholar   +1 more source

Patterns of Direct Oral Anticoagulant Use in Pediatric Patients: Results From a Multicenter National Database

open access: yesPediatric Blood &Cancer, Volume 72, Issue 8, August 2025.
ABSTRACT Background Direct oral anticoagulants (DOACs) have quickly gained favor due to oral availability, favorable risk profile, and reduced lab monitoring. This study aims to evaluate trends in DOAC use among pediatric patients with new venous thrombotic events (VTE) and factors associated with their selection.
Molly Mack   +2 more
wiley   +1 more source

Hemophilia B Gene Therapy with a High‐Specific‐Activity Factor IX Variant

open access: yesNew England Journal of Medicine, 2017
Lindsey A. George   +27 more
semanticscholar   +1 more source

Incidental Identification of Potentially Affected Individuals Through Expanded Carrier Screening During Preconception or Early Pregnancy

open access: yesPrenatal Diagnosis, Volume 45, Issue 9, Page 1151-1159, August 2025.
ABSTRACT Objective Expanded carrier screening (ECS) is used to assess the reproductive probability of having an offspring affected by an autosomal recessive or X‐linked recessive genetic disorder. Rarely, a presumably healthy individual may be identified to carry variants that could influence their health.
Yan Lü   +9 more
wiley   +1 more source

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