Results 31 to 40 of about 160,551 (221)

Experiencia clínica con medicamentos huérfanos para enfermedades raras metabólicas

Anales de Pediatría, 2022
Resumen: Introducción: Los errores congénitos del metabolismo son un grupo muy heterogéneo de enfermedades raras. La mayoría se pueden tratar con dieta y sustitución enzimática.Existen pocos pacientes y pocos estudios publicados en estas enfermedades ...
Alicia Caso-González, Jesica Núñez-Rodríguez, María-José Nebot-Villacampa, Yared González-Pérez, Raquel Marín-Gorricho, Claudia Leralta-González, Carmen Obaldia-Alaña   +6 more
doaj  

Lamellar Keratoplasty as a Method of Treatment Patients with Neurotrophic Keratopathy II and III Stages Outcome

Oftalʹmologiâ, 2022
Neurotrophic keratopathy (NTK) is an orphan, polyetiological disease that has recently been identified more frequently. Drug-resistant stages of NTC often require urgent surgical treatment.
M. Ezugbaya, I. A. Riks, S. V. Trufanov, S. Yu. Astakhov, S. S. Papanyan, L. K. Anikina, R. Boutaba, V. V. Gryaznova   +7 more
doaj   +1 more source

Evidence supporting regulatory-decision making on orphan medicinal products authorisation in Europe: methodological uncertainties

Orphanet Journal of Rare Diseases, 2018
Background To assess uncertainty in regulatory decision-making for orphan medicinal products (OMP), a summary of the current basis for approval is required; a systematic grouping of medical conditions may be useful in summarizing information and issuing ...
Caridad Pontes, Juan Manuel Fontanet, Roser Vives, Aranzazu Sancho, Mònica Gómez-Valent, José Ríos, Rosa Morros, Jorge Martinalbo, Martin Posch, Armin Koch, Kit Roes, Katrien Oude Rengerink, Josep Torrent-Farnell, Ferran Torres   +13 more
doaj   +1 more source

Identification of Tetraazacyclic Compounds as Novel Potent Inhibitors Antagonizing RORγt Activity and Suppressing Th17 Cell Differentiation. [PDF]

PLoS ONE, 2015
CD4+ T-helper cells that produce interleukin-17 (Th17 cells) are characterized as pathological T-helper cells in autoimmune diseases. Differentiation of human and mouse Th17 cells requires a key transcription regulator, retinoic acid receptor-related ...
Qingfeng Ding, Mei Zhao, Bolan Yu, Chuan Bai, Zhaofeng Huang   +4 more
doaj   +1 more source

DRUG FLURENISID AS AN IMMUNOMODULATOR FOR THE TREATMENT OF ORPHAN DISEASES

, 2017
Flurenizide was created as a result of purposeful synthesis of fl uorene series compounds and research into their chemical, physical, biological and pharmacological characteristics.
М. Kovalenko
semanticscholar   +1 more source

Trends of ex tempore drug preparation in Ukraine. Ways of their introduction into the practice

Infusion & Chemotherapy, 2020
Background. Advantages of the ex tempore formulation include the ability to provide the drug in the form and dosage, not available on the pharmaceutical market, but necessary for a particular patient; the ability to include the required ingredient in any
V.A. Heorhiiants
semanticscholar   +1 more source

Orphan drugs: Unmet societal need for non-profitable privately supplied new products

Research Policy, 2007
Due to the severity of rare diseases, the societal need for biopharmaceutical treatments for these diseases is high, despite low numbers of patients. Therefore, we investigated the barriers currently hindering the willingness to develop orphan drugs in the Netherlands.
Moors, E.H.M., Faber, J.
openaire   +4 more sources

Deciphering the pathway-specific regulatory network for production of ten-membered enediyne Tiancimycins in Streptomyces sp. CB03234-S

Microbial Cell Factories, 2022
Background The anthraquinone-fused 10-membered enediynes (AFEs), represented by tiancimycins (TNMs), possess a unique structural feature and promising potentials as payloads of antitumor antibody–drug conjugates.
Manxiang Zhu, Fan Zhang, Ting Gan, Jing Lin, Yanwen Duan, Xiangcheng Zhu   +5 more
doaj   +1 more source

Lycium barbarum Glycopeptide Alleviates Neomycin‐Induced Ototoxicity by Inhibiting Tryptophan Hydroxylase‐Mediated Serotonin Biosynthesis

Advanced Science, EarlyView.
Lycium barbarum glycopeptide (LBGP), which is further extracted from Lycium barbarum polysaccharides, exhibits significant protective effects against neomycin‐induced hearing dysfunction including oxidative stress in cochlea and loss of key cells in cochlea.
Yunhao Wu, Li Zhang, Shengda Cao, Jingwen Zhang, Cheng Li, Yunlong Shan, Qiuping Liu, Zhexiong Yu, Qiaojun Fang, Yuhua Zhang, Xiaolong Fu, Kwok‐Fai So, Renjie Chai   +12 more
wiley   +1 more source

Regulation of Antimycin Biosynthesis Is Controlled by the ClpXP Protease

mSphere, 2020
The survival of any microbe relies on its ability to respond to environmental change. Use of extracytoplasmic function (ECF) RNA polymerase sigma (σ) factors is a major strategy enabling dynamic responses to extracellular signals.
Bohdan Bilyk, Sora Kim, Asif Fazal, Tania A. Baker, Ryan F. Seipke   +4 more
doaj   +1 more source