Results 181 to 190 of about 136,469 (280)

Undetectable Hydroxyurea Levels in the Majority of Sickle Cell Disease Patients, Especially in Young Children

American Journal of Hematology, EarlyView.
ABSTRACT Hydroxyurea (HU) is the most widely prescribed disease‐modifying treatment in sickle cell disease (SCD), though treatment responses vary due to metabolism and adherence. We examined HU blood levels and treatment response in patients with homozygous sickle cell disease (HbSS).
Sigrid van der Veen, Bart J. Biemond, Marjon H. Cnossen, Pablo Bartolucci, Maria P. Boaro, Betzabel Cajiao Garcia, Raffaella Colombatti, M. D'Agnolo, Karin Fijnvandraat, Anna Collado Gimbert, Amira Idrizovic, Petros Kountouris, Mar Mañú‐Pereira, Elisabetta Mezzalira, Mariane de Montalembert, Erfan Nur, A. Pham Hung D' Alexandry D'Orengiani, Tiziana Sanavia, Saskia E. M. Schols, Marissa J. M. Traets, Minke A. E. Rab, Sara I. Reidel, Anita W. Rijneveld, Yosr Zaouali, Nanda M. Verhoeven‐Duif, Judith J. M. Jans, Eduard J. van Beers   +26 more
wiley   +1 more source

Clinical Outcomes and Patient Experiences With Celiprolol Therapy in Vascular Ehlers–Danlos Syndrome: The First Non‐European Cohort

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Vascular Ehlers–Danlos syndrome (vEDS) is a hereditary connective tissue disorder caused by heterozygous pathogenic variants in COL3A1. European studies have shown that celiprolol may reduce the risk of life‐threatening vascular events, but outcomes in non‐European populations and the therapy's psychological impact remain unclear. We conducted
Megumi Furuhata‐Yoshimura, Tomomi Yamaguchi, Tomoki Kosho   +2 more
wiley   +1 more source

Simple, robust and near optimal designs for the estimation of log-logistic dose response functions. [PDF]

Arch Toxicol
Holland-Letz T, Kopp-Schneider A.
europepmc   +1 more source

Milestone Attainment in Young Children With Arthrogryposis Multiplex Congenita: Developmental Profile and Associated Factors

American Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.
ABSTRACT Evidence on developmental milestones in children with arthrogryposis multiplex congenita (AMC) under the age of five is scarce. This multisite cross‐sectional study described developmental status and examined factors associated with milestone attainment in 143 children aged 0–66 months from a pediatric AMC Registry.
Ahlam Zidan, Sophia C. Pasia, Emmanouil Rampakakis, Reggie Hamdy, Frank Rauch, Lauren C. Hyer, Joel Lerman, Haluk Altiok, Krister Freese, Cary Mielke, Sarah B. Nossov, Philip F. Giampietro, Thania Ordaz‐Robles, Noémi Dahan‐Oliel   +13 more
wiley   +1 more source

Constructing targeted minimum loss/maximum likelihood estimators: a simple illustration to build intuition. [PDF]

Am J Epidemiol
Ross RK, Montoya LM, Goin DE, Díaz I, Renson A.   +4 more
europepmc   +1 more source

Early Versus Late Recurrence in Olfactory Neuroblastoma: A Multi‐Institutional Analysis of Predictive Risk Factors

International Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Olfactory neuroblastoma (ONB) exhibits variable recurrence patterns, with significant late recurrences occurring years after treatment. We investigated cutoff periods to define late recurrence and identified predictors that distinguish patients at risk of late recurrence from those considered cured.
Alexandria Harris, Anthony Tang, Nicholas Fung, Joao Paulo Almeida, Pierre‐Olivier Champagne, Juan Fernandez‐Miranda, Paul A. Gardner, Peter Hwang, Aristotelis Kalyvas, Chirag Patel, Zara M. Patel, Maria Peris Celda, Carlos D. Pinheiro‐Neto, Olabisi Sanusi, Carl H. Snyderman, Brian D. Thorp, Jamie J. Van Gompel, Georgios Zenonos, Nathan T. Zwagerman, Diana Bell, Edward C. Kuan, Eric W. Wang, Mathew Geltzeiler, Garret Choby   +23 more
wiley   +1 more source

Establishment and Validation of a Risk Prediction Model for the Superior Cluneal Nerve Entrapment Neuropathy after Thoracic or Lumbar Fracture: A Case-Comparative Study. [PDF]

J Pain Res
Liu XF, Ni ZM, Liu YL, Zhan DP, Zhang YF, Yin H.   +5 more
europepmc   +1 more source

Daily‐Life, Sensor‐Derived Tremor Measures Are Sensitive to Progression in Early Parkinson's Disease

Annals of Neurology, EarlyView.
Objective Sensitive outcome measures are critical for evaluating the efficacy of novel treatments for Parkinson's disease (PD). In this study, we assess the sensitivity to change of sensor‐derived daily‐life tremor measures over 2 years in unmedicated and medicated persons with early PD.
Nienke A. Timmermans, Ioan Gabriel Bucur, Diogo C. Soriano, Erik Post, Hayriye Cagnan, Sooyoon Shin, Max A. Little, Yordan P. Raykov, Bastiaan R. Bloem, Rick C. Helmich, Luc J.W. Evers   +10 more
wiley   +1 more source

Concurrent child undernutrition indicators in Malawi: application of trivariate logistic regression. [PDF]

Front Nutr
Kaombe TM.
europepmc   +1 more source

Pre‐Diagnostic Plasma Carotenoids, Tocopherols and Retinol Levels, and Risk of Amyotrophic Lateral Sclerosis

Annals of Neurology, EarlyView.
Objective Our goal was to examine whether pre‐diagnostic plasma carotenoids and tocopherols are associated with amyotrophic lateral sclerosis (ALS). Methods A nested case–control study within 4 United States cohorts, where 154 participants with pre‐diagnostic blood‐draw, were diagnosed during follow‐up with amyotrophic lateral sclerosis (ALS). Controls
Éilis J. O'Reilly, Kjetil Bjornevik, Jeremy D. Furtado, Alpa V. Patel, Ying Wang, Laurence N. Kolonel, Loïc Le Marchand, Alberto Ascherio   +7 more
wiley   +1 more source