Results 201 to 210 of about 16,177,862 (256)

Siblings With Duchenne Muscular Dystrophy: Exploring Diagnosis Age and Disease Progression in a Genetic Therapy‐Naïve Cohort

Annals of the Child Neurology Society, EarlyView.
ABSTRACT Introduction Duchenne muscular dystrophy (DMD) is the most common pediatric muscular dystrophy. Typically, there is a ~ 2‐year delay between symptom onset and diagnosis. Limited data on outcomes in early‐diagnosed individuals have limited the understanding of the clinical impact of early diagnosis.
Vaishnavi Brahmamdam, Cuixia Tian, Niki Armstrong, Valentina Pilipenko, Lisa J. Martin, Alexander Zygmunt, Irina Rybalsky, Lisa Reebals, Chinmayee B. Nagaraj   +8 more
wiley   +1 more source

A White Paper on Advancing Long‐Acting Therapeutics for Maternal and Pediatric Health by Bridging Gaps in Clinical Research, Access and Regulation

Clinical Pharmacology &Therapeutics, EarlyView.
As use cases for long‐acting therapeutics expand across clinical indications, there is a critical need to ensure the inclusion of women who are pregnant or breastfeeding, infants and children—populations with a historical gap in the availability of interventions already approved for use in adults.
Moherndran Archary, Robert Bies, Osei Boateng, Rachel Daley, Pierre Gashema, Mili Karina, Emily Njuguna, Sharon Nachman, Nathaniel Nkrumah, Carolyne Odula, Andrew Owen, Ethel D. Weld, Prajith Venkatasubramanian, Adeniyi Olagunju, on behalf of the Community of Practice for Long‐acting Therapeutics for Maternal and Paediatric Health, Rana Abutaima, Dorothy Akongo, Abdulnaser Alsharaa, Moherndran Archary, Shakir Atoyebi, Benoit Bestgen, Robert Bies, Osei Boateng, Andrew Butler, Edmund Capparelli, Rachel Daley, Layla Davies, Joelle Dountio, Henry Enzama, Pierre Gashema, Katila George, Dan Hawcutt, Rene Holm, Patrick Gad Iradukunda, Elodie Jambert, Mili Karina, Linda Lewis, Grace Miheso, Sebastien Morin, Andrew Morrison, Sharon Nachman, Shadia Nakalema, Emily Njunga, Nathaniel Nkrumah, Carolyne Odula‐Obonyo, Brenda Okware, Adeniyi Olagunju, Andrew Owen, Martina Penazzato, Natella Rakhmanina, Mphako Brighton Ratlabyana, Rachel Scott, Kimberly Struble, Andre‐Marie Tchouatieu, Catherine Unsworth, Prajith Venkatasubramanian, Catriona Waitt, Ethel Weld, Janine Winterbottom, Leena Zino   +59 more
wiley   +1 more source

Transcatheter-Based Palliation of Congenital Heart Disease in Very Low Birth Weight Infants. [PDF]

JACC Case Rep
Choi C.
europepmc   +1 more source

Model‐Informed Evaluation of Hydroxyurea Exposure During Lactation

Clinical Pharmacology &Therapeutics, EarlyView.
Hydroxyurea is a cornerstone therapy for sickle cell anemia; however, evidence guiding its use during lactation remains limited. This study aimed to develop a population pharmacokinetic (PK) model to characterize hydroxyurea disposition in maternal plasma and breast milk, and to quantify infant exposure under clinically relevant breastfeeding scenarios.
Anhar Hosawi, Kei Irie, Min Dong, Julie Ware, Russell Ware, Tomoyuki Mizuno   +5 more
wiley   +1 more source

Maternal Dipyrone Exposure and Risk of Major Congenital Malformations, Adverse Perinatal and Postnatal Outcomes: A Population‐Based Cohort Study

Clinical Pharmacology &Therapeutics, EarlyView.
Pain and fever are common in pregnancy. Dipyrone is widely used in Europe, Latin America, and parts of Asia, but is banned in other countries due to concerns about agranulocytosis. Evidence on its safety in pregnancy remains limited and inconsistent. This study aimed to evaluate whether maternal dipyrone use during the first‐ and third‐trimesters is ...
Itamar Ben Shitrit, Daphna Idan, Ariel Avraham Hasidim, Tal Michael, Amalia Levy, Gali Pariente, Eitan Lunenfeld, Sharon Daniel   +7 more
wiley   +1 more source

Clinical outcomes and longitudinal growth trajectories in preterm infants fed donor human milk versus formula: A retrospective cohort study. [PDF]

Medicine (Baltimore)
Youn SE, Youn SE, Kim TH, Chung SH.
europepmc   +1 more source

Long‐Acting Therapeutics in Pediatric Health: Bridging Innovation and Access

Clinical Pharmacology &Therapeutics, EarlyView.
Children have distinct therapeutic needs arising from age‐dependent physiology, disease epidemiology, formulation requirements, dosing considerations, and safety vulnerabilities. Despite substantial reductions in childhood mortality over the past three decades, nearly 5 million children under five died in 2023, with infectious diseases continuing to ...
Prajith Venkatasubramanian, Moherndran Archary, Daniel B. Hawcutt, Rachel Daley, Edmund Capparelli, Adeniyi Olagunju   +5 more
wiley   +1 more source

Pharmacokinetics of Rifapentine and Desacetyl Rifapentine in Maternal Plasma and Breast Milk & Infant Cord Blood and Plasma: A Model‐Based Analysis of Mother–Infant Pairs for Tuberculosis Prevention

Clinical Pharmacology &Therapeutics, EarlyView.
IMPAACT 2001 (NCT02651259) evaluated the safety and pharmacokinetics (PK) of once‐weekly isoniazid and rifapentine in pregnant and postpartum women for tuberculosis prevention. Rifapentine (RPT) and desacetyl rifapentine (desRPT) PK data were collected in maternal plasma, breast milk, infant cord blood, and infant plasma.
Priya Jayachandran, Jyoti S. Mathad, Nan Zhang, Paula Britto, Lubbe Wiesner, Grace Montepiedra, Jennifer Norman, Ellen Townley, Sarah Bradford, Sandesh Patil, Stephanie Popson, Tsungai Chipato, Vanessa Rouzier, Deborah Langat, Amphan Chalermchockcharoentkit, Amita Gupta, Kelly E. Dooley, Radojka Savic, on behalf of the IMPAACT 2001 Study Team   +18 more
wiley   +1 more source

Model‐Informed Dose Optimization of Anakinra in Preterm Neonates Implications for Safe and Effective Dosing

Clinical Pharmacology &Therapeutics, EarlyView.
Anakinra, an interleukin‐1 receptor antagonist, shows promise for reducing inflammatory complications of premature birth. However, the pharmacokinetics (PK) of anakinra in neonates has not been characterized, limiting evidence‐based dosing. This study aimed to develop a population PK model for anakinra to guide dosing in preterm neonates.
Jia Li, David K. Metz, Elys Green, Gergely Toldi, Robert Galinsky, Megan Clark, Rebecka Atkinson, Ina Rudloff, Josephine C. Owen, Sara K. DiSimone, Samuel C. Forster, Steven X. Cho, Atif Majid, Graeme R. Polglase, Marcel Favilla, Pramod Pharande, Atul Malhotra, Calum T. Roberts, Scott H. Stansfield, Arvind Sehgal, Kenneth Tan, Alistair J. Gunn, Philip J. Berger, Alex Veldman, Rod W. Hunt, Claudia A. Nold‐Petry, Marcel F. Nold, Carl M. J. Kirkpatrick   +27 more
wiley   +1 more source