Results 121 to 130 of about 23,786 (267)

Beyond the label: Rethinking off‐label drug use in paediatrics. Towards a scientifically grounded and safer future for paediatric pharmacotherapy

British Journal of Clinical Pharmacology, EarlyView.
Abstract Despite regulatory progress being made in the past two decades, off‐label drug use in paediatrics remains pervasive, with prevalence estimated between 3% and 97% of prescriptions across different clinical settings. Off‐label use—defined as prescribing outside the conditions described in the Summary of Product Characteristics (SmPC)—is often ...
Tjitske M. van der Zanden, Miriam G. Mooij, Matthijs de Hoog, Saskia N. de Wildt   +3 more
wiley   +1 more source

Glycoengineered Recombinant Alpha1‐Antitrypsin Results in Comparable In Vitro and In Vivo Activities to Human Plasma‐Derived Protein

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Alpha‐1‐antitrypsin (A1AT) is a multifunctional, clinically important, high‐value therapeutic glycoprotein that can be used for the treatment of many diseases, such as A1AT deficiency, diabetes, graft‐versus‐host disease, cystic fibrosis, and various viral infections. Currently, the only U.S. food and drug administration‐approved treatment for
Frances Rocamora, Chen‐Lin Hsieh, Sanne Schoffelen, Johnny Arnsdorf, Eric A. Toth, Abdul S. Yunus, Thomas E. Cleveland IV, Sara Petersen Bjørn, Mina Ying Min Wu, Noel G. McElvaney, Bjørn Gunnar Rude Voldborg, Thomas R. Fuerst, Nathan E. Lewis   +12 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

The Pediatric Growth Hormone Deficiency Patient Journey: Identifying Opportunities for Digital Health Interventions [PDF]

Pediatric growth hormone deficiency (PGHD) is a chronic condition where the pituitary gland fails to produce sufficient growth hormone, leading to delayed growth and developmental challenges.
Giunti, Guido, Halabi, Ammar, Dimitri, Paul, Koledova, Ekaterina, Harvey, Jamie, Michelis, Fulvio   +5 more
core   +1 more source

Payload‐Based Clinical Pharmacology Review of Approved Antibody–Drug Conjugates: Commonalities and Considerations for Streamlined Development

Clinical Pharmacology &Therapeutics, EarlyView.
Antibody–drug conjugates (ADCs) combine the specificity of an antibody with the potency of a cytotoxic drug. Thirteen ADCs, utilizing seven unique cytotoxic payloads and targeting 11 distinct antigens, are currently approved by the US Food and Drug Administration as of June 2025, representing a rapidly growing and highly promising class of anticancer ...
Sijie Lu, Qingqing Xiao, Lixuan Qian, Siyuan Sun, Zhu Zhou, Michael Z. Liao, Chunze Li   +6 more
wiley   +1 more source

Maternal and Neonatal Complications Associated with Breast Cancer Systemic Treatments—A VigiBase Disproportionality Analysis Study

Clinical Pharmacology &Therapeutics, EarlyView.
Pregnancy‐associated breast cancer (PrBC) presents therapeutic challenges. Understanding maternal–fetal safety of systemic anticancer therapies is critical. We performed a case/non‐case disproportionality analysis using the WHO global pharmacovigilance database up to January 2024, to evaluate maternal–fetal outcomes associated with breast cancer (BC ...
Rayan Kabirian, Floriane Jochum, Elise Dumas, Florence Coussy, Kevin Bihan, Bénédicte Lebrun‐Vignes, Barbara Pistilli, Marc‐Antoine Benderra, Lise Selleret, Laurent Chouchana, Fabien Reyal, Anne‐Sophie Hamy, Paul Gougis   +12 more
wiley   +1 more source

Expression of mutant TIE2 p.L914F during mouse development causes embryonic lethality and defects in vascular remodeling

Developmental Dynamics, EarlyView.
Abstract Background Sporadic venous malformation (VM) is associated with the hyperactivating p.L914F mutation in TIE2, a receptor tyrosine kinase essential for vascular development. This mutation is not found in hereditary VM, suggesting incompatibility with life when expressed during early vascular development.
Lindsay J. Bischoff, Sandra Schrenk, Kara Soroko, Chhiring Sherpa, Ashok Arasu, Damien Reynaud, Elisa Boscolo   +6 more
wiley   +1 more source

Treatment with long-acting growth hormone: effectiveness and safety

Frontiers in Endocrinology
Long-acting growth hormone (GH) formulations represent a clinically effective alternative to daily recombinant human GH (rhGH) administration, enabling extended dosing intervals and enhancing patient adherence. This approach is particularly beneficial in
Livia Basile, Rossella Cannarella, Rosita A. Condorelli, Francesco Frasca, Aldo E. Calogero, Sandro La Vignera   +5 more
doaj   +1 more source

Micronutrient Status in Children Suffering From Anorexia Nervosa: A Cohort of 349 Patients in a Referral Center for Eating Disorders

International Journal of Eating Disorders, EarlyView.
ABSTRACT Objective Anorexia nervosa (AN) is a serious illness in which more than half of all deaths are due to malnutrition. Critically low energy and protein intake are known causes of massive weight loss, whereas micronutrient deficiencies due to a low‐calorie food pattern remain poorly characterized in children with AN. Micronutrient deficiencies in
Zenaida Iordan, Diane Morfin, Marion Bernetiere, Marie Fauchet, Noël Peretti, Bérénice Segrestin, Pierre Poinsot   +6 more
wiley   +1 more source

Retinal Nerve Fiber Layer Thickness in Children With Isolated Growth Hormone Deficiency [PDF]

Isolated growth hormone deficiency (iGHD) is a condition causing severe short stature due to a lack of growth hormone (GH). GH\u27s impact on the eye, particularly retinal nerve fiber layer (RNFL), is unclear.
Gulcenur Ozturan; Digdem Bezen; Gulderen Karaca; Dogancan Ozturan; Mustafa Nuri Elcioglu
core