Results 241 to 250 of about 149,441 (303)

OGFRL1 deficiency causes CRMO via pathological osteoclastogenesis, with therapeutic response to TNF inhibitor

Arthritis &Rheumatology, Accepted Article.
Objectives To verify the pathogenesis of the OGFRL1 loss‐of‐function variant (c.30del, p. F10Ffs*110) identified in a CRMO patient and investigate the underlying mechanism. Methods Whole exome sequencing and Sanger sequencing were performed to identify and confirm the variant.
Wen Xiong, Yusha Wang, Tingyan He, Jinjian Fu, Ying Luo, Jinbo Wang, Jun Wang, Xiaomin Yu, Qing Zhou, Jun Yang   +9 more
wiley   +1 more source

Switching disease‐modifying therapies in patients with spinal muscular atrophy: A systematic review on effectiveness outcomes

British Journal of Clinical Pharmacology, EarlyView.
With multiple disease‐modifying therapies now available, treatment switching has become an important clinical consideration in the management of spinal muscular atrophy (SMA). While some switches are prompted by suboptimal clinical response, more commonly they are driven by treatment burden, convenience, or adverse events.
Andrej Belančić, Elvira Meni Maria Gkrinia, Patrick Eustaquio, Andrea Katrin Faour, Dinko Vitezić   +4 more
wiley   +1 more source

Clinical Images: Vacuoles, E1 enzyme, X‐linked, autoinflammatory, somatic syndrome presenting as aseptic pustular dermatosis: A rare clinical manifestation

Arthritis &Rheumatology, EarlyView.
Peter Chen, Héloïse Briançon, Deborah Eshagh, Tali‐Anne Szwebel, Benjamin Terrier, Pierre Sohier, Rudy Birsen   +6 more
wiley   +1 more source

Navigating adverse immunostimulation: A practical guide for clinical researchers

British Journal of Clinical Pharmacology, EarlyView.
Problem Setting As drug development moves towards more complex products, early clinical development programmes are increasingly hampered by unwanted and/or unexpected activation of the immune system (adverse immune stimulation, AIS). Solution At the Centre for Human Drug Research, we have introduced standardized procedures to make AIS manageable, while
Juliette A. van den Noort, Anouk C. Meijs, Naomi B. Klarenbeek, Matthijs Moerland   +3 more
wiley   +1 more source

Induction of antigen-specific suppressor cells in patients with hay fever receiving immunotherapy

, 1985
Hiroshi Nagaya
openalex   +1 more source

Adverse immunostimulation in early phase clinical trials: Key findings and recommendations based on the investigator's clinical experience

British Journal of Clinical Pharmacology, EarlyView.
Problem setting The emergence of therapeutic proteins has coincided with an increase of acute adverse immunostimulation (AIS). AIS has occured in clinical trials despite compliance with regulatory guidelines on preclinical evaluation and its incidence is anticipated to increase even further.
Juliette A. van den Noort, Marella C. E. van Ruissen, Lisanne C. A. Smidt, Naomi B. Klarenbeek, Philip H. K. Kremer, Manon A. A. Jansen, Jacobus Burggraaf, Joop M. A. van Gerven, Matthijs Moerland   +8 more
wiley   +1 more source

Tell me about your hay fever: a qualitative investigation of allergic rhinitis management from the perspective of the patient. [PDF]

NPJ Prim Care Respir Med, 2018
Cvetkovski B, Kritikos V, Yan K, Bosnic-Anticevich S.   +3 more
europepmc   +1 more source

Safety, tolerability and pharmacokinetics of DNDI‐6148, a novel agent for leishmaniasis: A randomized, controlled, single ascending dose study in healthy participants

British Journal of Clinical Pharmacology, EarlyView.
Aim The benzoxaborole derivative DNDI‐6148 is an antiparasitic agent with activity against multiple Leishmania protozoan species, including L. infantum and L. donovani, which cause visceral leishmaniasis. We investigated the safety, tolerability and pharmacokinetics of single oral doses of DNDI‐6148 in a randomized, parallel‐group, placebo‐controlled ...
Jean‐Yves Gillon, Sophie Delhomme, Delphine Launay, Séverine Blesson, Stéphanie Braillard, Pegah Maghdooni, Frauke Assmus, Richard Hoglund, Joel Tarning, Sabrina Loyau, Byron Arana, Mathilde Latreille‐Barbier, Yves Donazzolo   +12 more
wiley   +1 more source

Paediatric development of radiopharmaceutical imaging agents and radioligand therapeutics

British Journal of Clinical Pharmacology, EarlyView.
Abstract This review focuses on the development of radiopharmaceutical imaging agents and radioligand therapeutics for paediatric use. Nuclear medicine plays an important role in the diagnosis and treatment of various childhood conditions, including cancers, infections and brain disorders.
Justin L. Hay, Amandine Manon, Hunter Stephens, S. Y. Amy Cheung, Bart de Keizer, Erik T. te Beek   +5 more
wiley   +1 more source

Population pharmacokinetics and dose–response relationships of mitoxantrone in children with acute myeloid leukaemia

British Journal of Clinical Pharmacology, EarlyView.
Background Information on mitoxantrone pharmacokinetics in children is lacking and reduced dosing regimens applied to infants are supported by limited scientific rationale. The current study characterized mitoxantrone pharmacokinetics in a childhood acute myeloid leukaemia patient population and provides a data‐informed assessment of dosing.
Andrew M. Brandon, Hinke Huisman‐Siebinga, Shelby Barnett, Paul Wetherell, Pamela Kearns, Brenda Gibson, Nicholas Heaney, Owen Smith, André Baruchel, Arnaud Petit, Andrew Moore, Kayode Ogungbenro, Alwin D. R. Huitema, Gareth J. Veal   +13 more
wiley   +1 more source