Results 121 to 130 of about 49,420 (307)

The Clinical Spectrum and Neurodevelopmental Pathogenesis of KPTN‐Related Disorder in a Mouse Model

Annals of Neurology, EarlyView.
Objective Pathogenic variants in Kaptin (KPTN) cause KPTN‐related disorder (KRD). KPTN modulates mTOR signaling activation within the KICSTOR complex in response to cellular amino acid levels. We define the clinical spectrum and investigate the developmental pathogenesis of KRD.
Lettie E. Rawlins, Philip H. Iffland II, John Page, Rebecca Z. Flessner, Soad M. Elziny, Irina Sbornova, Janice K. Babus, Sophie R. Bruckmeier, Ria Parikh, Merel Verhoeven, James Fasham, Joseph S. Leslie, Richard Caswell, Nishanka Ubeyratna, Olivia Wenger, Ethan M. Scott, John Schreiber, Steffen Syrbe, Annick Klabunde‐Cherwon, Martina Owens, Andrew H. Crosby, Emma L. Baple, Peter B. Crino, the KPTN Consortium, Andrea Seeley, Heather Rocha, Sabine Rudnick, Ulrich Schaatz, Tobias Haack, Eva Schwaibold, Caleb Bupp, Klaas Wierenga, John Schreiber, Thorkild Terkelsen, Sarina Kant, J. C. Herkert, Michael Wright, Boris Keren, Solveig Heide, Pamela Jackson, Pradeep Vasudevan, Helen Stewart, Perrine Charles, Andreas Hahn, Saskia Biskup, Inger‐Lise Mero, Martin Heier, Conny van Ravenswaaij‐Arts, Esther Nibbeling, Stephanie Efthymiou, Erum Afzal, Ian Ellis, Raza Maroofian, Almundher Al‐Maawali, David Coman, Anthony Morosini, Sajjad Biglari, Pooneh Nikuei, Saadet Mercimek‐Andrews, Prab Prabhakar, Raymond Louie, Kameryn Butler, Olivia Wenger, Ethan M. Scott, Andrea Seeley, Heather Rocha, Amber Begtrup, Sabine Rudnik, Ulrich Schatz, Tobias Haack, Eva Schwaibold, Caleb Bupp, Klaas Wierenga, John Schreiber, Thorkild Terkelsen, Sarina Kant, J. C. Herkert, Michael Wright, Boris Keren, Solveig Heide, Pamela Jackson, Pradeep Vasudevan, Helen Stewart, Perrine Charles, Andreas Hahn, Saskia Biskup, Inger‐Lise Mero, Martin Heier, Marijn Stokman, Conny van Ravenswaaij‐Arts, Esther Nibbeling, Stephanie Efthymiou, Erum Afzal, Kristina Zhelcheska, Henry Houlden, Ian Ellis, Reza Maroofian, Gholamreza Shariati, Hamid Galehdari, Almundher Al‐Maawali, Adila Al‐Kindi, Andrew Y. Shuen, Victoria Siu, Annick Klabunde‐Cherwon, Steffen Syrbe   +104 more
wiley   +1 more source

Nasal powder formulation employing microenvironmental pH-modifier for rapid absorption of mirtazapine

Pharmaceutical Science Advances
Antidepressant mirtazapine (MRZ) has been drawing attention in the management of delirium. However, oral use of MRZ could have drawbacks in onset of actions and ease of administration by caregivers.
Kohei Yamada, Tsubasa Kihara, Kaori Shinsato, Hirofumi Yasui, Michihiro Shino, Hideyuki Sato, Satomi Onoue   +6 more
doaj   +1 more source

Review of medicine registration system in Tanzania [PDF]

, 2019
Philosophiae Doctor - PhDRegistration of medicines is an essential function of the national medicine regulatory system of any country. Since the start of the medicine registration system in Tanzania in early 2000s, no systematic review has been carried
KISOMA, SUNDAY
core  

A Randomized, Placebo‐Controlled Trial of Hydroxychloroquine in Incomplete Lupus

Arthritis &Rheumatology, EarlyView.
Objective Patients with features of systemic lupus erythematosus (SLE) who do not fulfill classification criteria can be designated as incomplete lupus erythematosus (ILE). This condition includes individuals with a high risk of progression to SLE. Treatment of ILE may reduce symptoms, severity, and incidence of SLE.
Nancy J. Olsen, Duanping Liao, Judith A. James, Joel M. Guthridge, Cristina Arriens, Diane Kamen, Mariko Ishimori, Daniel J. Wallace, Christopher Striebich, Sonali Narain, Benjamin F. Chong, Fan He, Eric W. Schaefer, Vernon M. Chinchilli, David R. Karp   +14 more
wiley   +1 more source

Interleukin‐5, Eosinophils, and Interleukin‐5 Pathway Inhibitors in Eosinophilic Granulomatosis With Polyangiitis

Arthritis &Rheumatology, EarlyView.
Interlukin‐5 (IL‐5) plays a crucial role in the pathogenesis of eosinophilic granulomatosis with polyangiitis (EGPA) by promoting eosinophil differentiation, activation, and survival. We present here a typical case of EGPA in which treatment with IL‐5 pathway inhibitors is prescribed, showing to be beneficial for the patient.
Alvise Berti, Christian Pagnoux
wiley   +1 more source

The effect of mepolizumab dosage form on treatment outcomes in severe asthma

Frontiers in Medicine
BackgroundA monoclonal antibody such as mepolizumab typically first appears as a parenteral lyophilized formulation (LYO), then as various parenteral solution forms, and finally as a self-administered form at homecare.
Martina Vetchá, Kateřina Kubová, Constantinos Glynos, Sylvie Pavloková, Irena Krčmová, Irena Krčmová, Eva Voláková, Eva Voláková, Ondřej Fibigr, Ondřej Fibigr, Beáta Hutyrová, Beáta Hutyrová, Alena Vlachová, Jiří Zeman, David Vetchý   +14 more
doaj   +1 more source

The Effect of Intranasal Niclosamide on Nasal Symptoms in Patients with Antineutrophil Cytoplasmic Antibody–Associated Vasculitis

Arthritis &Rheumatology, EarlyView.
Objective Ear, nose, and throat (ENT) manifestations are common in antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis (AAV). There is an unmet need for drugs to target these manifestations. Granuloma formation is characteristic of proteinase 3 (PR3)–AAV. In a zebrafish model, niclosamide inhibits PR3‐induced granuloma formation.
Benjamin Lim, Wendi Qian, Francis Dowling, Alan Salama, Rona M Smith   +4 more
wiley   +1 more source

Evaluation of Drug Permeation Enhancement by Using In Vitro and Ex Vivo Models

Pharmaceuticals
Drugs administered by means of extravascular routes of drug administration must be absorbed into the systemic circulation, which involves the movement of the drug molecules across biological barriers such as epithelial cells that cover mucosal surfaces ...
Johan D. Steyn, Anja Haasbroek-Pheiffer, Wihan Pheiffer, Morné Weyers, Suzanne E. van Niekerk, Josias H. Hamman, Daniélle van Staden   +6 more
doaj   +1 more source

Prediction of Relapse and Glucocorticoid Dependence in Eosinophilic Granulomatosis With Polyangiitis: Findings From a Large European Cohort

Arthritis &Rheumatology, EarlyView.
Objective Eosinophilic granulomatosis with polyangiitis (EGPA) is a small vessel vasculitis characterized by eosinophilia, asthma, and ear, nose, and throat (ENT) involvement. Although glucocorticoids (GCs) are effective in controlling symptoms, relapses and GC dependence are common. The aim of this study was to develop predictive models for vasculitis
Matthias Papo, Pauline Martinot, Renato A. Sinico, Vítor Silvestre‐Teixeira, Nils Venhoff, Maria‐Letizia Urban, Michele Iudici, Juliane Mahrhold, Francesco Locatelli, Giulia Cassone, Franco Schiavon, Benjamin Seeliger, Thomas Neumann, Claudia Feder, Matthieu Groh, Chiara Marvisi, Maxime Samson, Thomas Barba, David Jayne, Arianna Troilo, Jens Thiel, Bernhard Hellmich, Sara Monti, Carlomaurizio Montecucco, Carlo Salvarani, Jean‐Emmanuel Kahn, Bernard Bonnotte, Cécile‐Audrey Durel, Xavier Puéchal, Luc Mouthon, Loïc Guillevin, Giacomo Emmi, Augusto Vaglio, Raphaël Porcher, Benjamin Terrier, on behalf of the French Vasculitis Study Group and the Eosinophilic Granulomatosis with Polyangiitis European Study Group   +35 more
wiley   +1 more source

The Role of Microbiota Metabolites Propionic Acid, p‐Cresol, and 4‐Ethylphenyl Sulfate in Autism Susceptibility: A Systematic Review

Autism Research, EarlyView.
ABSTRACT The etiopathogenesis of Autism Spectrum Disorder (ASD) encompasses complex interactions between genetic and environmental risk factors. The high prevalence of gastrointestinal disorders in autistic individuals has propelled a growing interest in the possible involvement of gut dysbiosis in ASD pathogenesis.
Laura Sandoni, Lisa Asta, Nicole Giompaolo, Rinvil Renaldi, Alberto Amaretti, Maddalena Rossi, Antonio M. Persico   +6 more
wiley   +1 more source