Results 41 to 50 of about 7,438 (233)

The European challenges of funding orphan medicinal products [PDF]

, 2018
Funding of orphan medicinal products (OMPs) is an increasing challenge in the European Union (EU).To identify the different methods for public funding of OMPs in order to map the availability for rare disease patients, as well as to compare the public ...
Arickx, Francis, Bucsics, Anna, Cohn-Zanchetta, Emanuelle, Fürst, Jurij, Kaló, Zoltán, Kamusheva, Maria, Kawalec, Pawel, Molnár, Mária Judit, Petrova, Guenka, Slaby, Juraj, Stawowczyk, Ewa, Szegedi, Márta, Vocelka, Milan, Zechmeister-Koss, Ingrid, Zelei, Tamás   +14 more
core   +4 more sources

High‐throughput markerless pose estimation and home‐cage activity analysis of tree shrew using deep learning

Animal Models and Experimental Medicine, EarlyView.
We present a deep learning approach to achieve markerless pose estimation and recognize multiple spontaneous behaviors of tree shrews. This high‐throughput approach can monitor the home‐cage activities of 16 tree shrews simultaneously over an extended period.
Yangzhen Wang, Feng Su, Rixu Cong, Mengna Liu, Kaichen Shan, Xiaying Li, Desheng Zhu, Yusheng Wei, Jiejie Dai, Chen Zhang, Yonglu Tian   +10 more
wiley   +1 more source

Safety, tolerability, pharmacokinetics and pharmacodynamics of HSK31858, a novel oral dipeptidyl peptidase‐1 inhibitor, in healthy volunteers: An integrated phase 1, randomized, double‐blind, placebo‐controlled, single‐ and multiple‐ascending dose study

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim Dipeptidyl peptidase‐1 (DPP‐1) inhibitors have been studied for the treatment of neutrophil‐mediated inflammatory diseases including bronchiectasis, bronchial asthma and cystic fibrosis. This study evaluated the pharmacokinetics, pharmacodynamics, safety and tolerability of DPP‐1 inhibitor HSK31858 in healthy Chinese volunteers.
Yuhao Wang, Chao Yu, Mengyue Hu, Lu Wang, Meixia Chen, Hanmo Liu, Nan Wu, Jie Hou   +7 more
wiley   +1 more source

Preparation of a Selective L-Phenylalanine Imprinted Polymer Implicated in Patients with Phenylketonuria

Iranian Journal of Medical Sciences, 2014
Background: Molecular imprinting is a method for synthesizing polymers with structure-selective adsorption properties with applications such as, selectivity binding, drug delivery systems and anti-bodies.
Parvaneh Najafizadeh, Soltan Ahmad Ebrahimi, Mohammad Reza Panjehshahin, Seyed Mahdi Rezayat Sorkhabadi   +3 more
doaj  

Inherited metabolic epilepsies–established diseases, new approaches

Epilepsia Open, EarlyView.
Abstract Inherited metabolic epilepsies (IMEs) represent the inherited metabolic disorders (IMDs) in which epilepsy is a prevailing component, often determining other neurodevelopmental outcomes associated with the disorder. The different metabolic pathways affected by individual IMEs are the basis of their rarity and heterogeneity.
Itay Tokatly Latzer, Phillip L. Pearl
wiley   +1 more source

Urinary neopterin concentrations vs total neopterins for clinical utility [PDF]

, 1989
Fuchs D, Milstien S, Krämer A, et al. Urinary neopterin concentrations vs total neopterins for clinical utility. Clinical Chemistry. 1989;35(12):2305-2307.Neopterin measurements are especially useful as an early marker in (e.g.) allograft rejections and ...
Fuchs, Dietmar, Goedert, James J., Kaufman, Seymour, Krämer, Alexander , Milstien, Sheldon, Reibnegger, Gilbert, Wachter, Helmut, Werner, Ernst R.   +7 more
core   +1 more source

Identification of a Novel Mutation in the PAH Gene in an Iranian Phenylketonuria Family: A Case Report

Iranian Journal of Public Health, 2017
Phenylketonuria (PKU) is an inborn error of amino acid metabolism with an autosomal recessive inheritance caused in most cases by mutations in the phenylalanine hydroxylase (PAH) gene. PKU has wide allelic heterogeneity.
Masoumeh RAZIPOUR, Daniz KOOSHAVAR, Elaheh ALAVINEJAD, Seyede Zahra SAJEDI, Neda MOHAJER, Aria SETOODEH, Saeed TALEBI, Mohammad KERAMATIPOUR   +7 more
doaj  

The influence of microbiome‐derived amino acids metabolites in shaping the glioma immunosuppressive microenvironment

Interdisciplinary Medicine, EarlyView.
Left: The disruption of the balance between carcinogenic bacteria and probiotics mediated by antibiotics, protein, and glucose intake. Middle: The gut microbiota‐derived amino acids, primarily through tryptophan, tyrosine, arginine, and branched‐chain amino acid pathways, regulate glioma development via mechanisms such as AHR activation, metabolic ...
Qianquan Ma, Zhihao Song, Chenlong Yang, Zijin Zhao, Guodong Tang, Jia You, Chong Zeng, Jun Yang, Qing Liu, Haoyu Li, Wei Huang   +10 more
wiley   +1 more source

Engineered strategies for the allergic disease therapy

Interdisciplinary Medicine, EarlyView.
Allergic diseases exist widely across the globe and now there are very limited therapeutics for the treatment of these diseases. In this review, we summarize the recent advances in engineering strategies including nanoemulsion and hydrogel to improve the therapeutic efficacy of allergen‐specific immunotherapy with decreased side effects and long‐term ...
Yun Tian, Xin Wu, Xiaoqi Sun, Lei Rong, Kai Han   +4 more
wiley   +1 more source

Breastfeeding follow-up in the treatment of children with phenylketonuria [PDF]

A fenilcetonúria (PKU) ocorre na incapacidade para transformar fenilalanina em tirosina, trazendo efeitos tóxicos para o sistema nervoso central. Tradicionalmente, no tratamento da PKU, o aleitamento materno é substituído por fórmula láctea.
Anastácio-Pessan, Fernanda da Luz, Caldeira, Ana Cláudia Gandara Casarin, Gejão, Mariana Germano, Lamônica, Dionísia Aparecida Cusin, Pedro, Karla Panice, Rolim-Liporacci, Maura Contieri, Stump, Marisdalva Viegas   +6 more
core   +1 more source