Pathogenic Germline PALB2 and RAD50 Variants in Patients With Relapsed Ewing Sarcoma
Pediatric Blood &Cancer, EarlyView.ABSTRACT Approximately 10% of patients with Ewing sarcoma (EwS) have pathogenic germline variants. Here, we report two cases: first, a novel germline pathogenic variant in partner and localizer of BRCA2 (PALB2) in a patient with a late EwS relapse. Its impact on homologous recombination is demonstrated, and breast cancer risk is discussed.
Molly Mack +12 more
wiley +1 more source
Extension of the asymptotic relative efficiency method to select the primary endpoint in a randomized clinical trial [PDF]
, 2014 We extend the ARE method proposed in Gómez and Lagakos (2013) devised to decide which primary endpoint to choose when comparing two treatments in a randomized clinical trial.
Gómez Melis, Guadalupe +1 more
core
A comparison of methods for treatment selection in seamless phase II/III clinical trials incorporating information on short-term endpoints [PDF]
, 2015 In an adaptive seamless phase II/III clinical trial interim analysis data are used for treatment selection, enabling resources to be focussed on comparison of more effective treatment(s) with a control.
Friede, Tim +4 more
core +1 more source
Phosphatidylinositol 4‐kinase as a target of pathogens—friend or foe?
FEBS Letters, EarlyView.This graphical summary illustrates the roles of phosphatidylinositol 4‐kinases (PI4Ks). PI4Ks regulate key cellular processes and can be hijacked by pathogens, such as viruses, bacteria and parasites, to support their intracellular replication. Their dual role as essential host enzymes and pathogen cofactors makes them promising drug targets.
Ana C. Mendes +3 more
wiley +1 more source
Alzheimer’s Research & Therapy, 2020 Background There is growing interest in identifying sensitive composite cognitive tests to serve as primary endpoints in preclinical Alzheimer’s disease (AD) treatment trials.
Jessica B. Langbaum +10 more
doaj +1 more source
The effects of donepezil in Alzheimer's disease - Results from a multinational trial [PDF]
, 1999 Donepezil has been shown to be well tolerated and to improve cognition and global function in patients with mild to moderately severe Alzheimer's disease (AD).
Burns, A. +7 more
core +1 more source
The Caenorhabditis elegans DPF‐3 and human DPP4 have tripeptidyl peptidase activity
FEBS Letters, EarlyView.The dipeptidyl peptidase IV (DPPIV) family comprises serine proteases classically defined by their ability to remove dipeptides from the N‐termini of substrates, a feature that gave the family its name. Here, we report the discovery of a previously unrecognized tripeptidyl peptidase activity in DPPIV family members from two different species.
Aditya Trivedi, Rajani Kanth Gudipati
wiley +1 more source
Peptide‐based ligand antagonists block a Vibrio cholerae adhesin
FEBS Letters, EarlyView.The structure of a peptide‐binding domain of the Vibrio cholerae adhesin FrhA was solved by X‐ray crystallography, revealing how the inhibitory peptide AGYTD binds tightly at its Ca2+‐coordinated pocket. Structure‐guided design incorporating D‐amino acids enhanced binding affinity, providing a foundation for developing anti‐adhesion therapeutics ...
Mingyu Wang +9 more
wiley +1 more source
Clinical Trial Risk in Non-Hodgkin’s Lymphoma: Endpoint and Target Selection
Journal of Pharmacy & Pharmaceutical Sciences, 2011 Purpose. To quantify the clinical trial risk of new drug development in Non-Hodgkin’s lymphoma (NHL). Risk estimates for this disease have not been reported before. Methods.
Jayson Lee Parker +2 more
doaj +1 more source
Noninvasive vagus nerve stimulation as acute therapy for migraine. The randomized PRESTO study [PDF]
, 2018 Objective: To evaluate the efficacy, safety, and tolerability of noninvasive vagus nerve stimulation (nVNS; gammaCore; electroCore, LLC, Basking Ridge, NJ) for the acute treatment of migraine in a multicenter, double-blind, randomized, sham-controlled ...
Ambrosini, Anna +12 more
core +2 more sources