Results 61 to 70 of about 3,868,024 (353)

Clinical pharmacology information in regulatory submissions and labeling: A comparative analysis of orphan and non‐orphan drugs approved by the FDA

open access: yesClinical and Translational Science, 2022
Clinical pharmacology is an integral discipline supporting the development, regulatory evaluation, and clinical use of drugs for the treatment of both common and rare diseases.
Julie Hsieh   +4 more
doaj   +1 more source

Accelerating orphan drug development [PDF]

open access: yesNature Reviews Drug Discovery, 2010
Interest in developing drugs for rare diseases has increased substantially in recent years. This article from the US Food and Drug Administration highlights the role of regulators in catalysing further progress in this field.
Coté, Timothy R.   +2 more
openaire   +3 more sources

Physiopathological Implications of 7TM Receptors [PDF]

open access: yes, 2010
Seven-transmembrane (7TM) receptors are one of the most important proteins involved in perception of extracellular stimuli and regulation of variety of intracellular signaling pathways.
Cygankiewicz, Adam
core   +1 more source

A multilayer network approach for guiding drug repositioning in neglected diseases [PDF]

open access: yes, 2016
Drug development for neglected diseases has been historically hampered due to lack of market incentives. The advent of public domain resources containing chemical information from high throughput screenings is changing the landscape of drug discovery for
Berenstein, Ariel José   +3 more
core   +3 more sources

Impacts of adjustment of National Reimbursement Drug List on orphan drugs volume and spending in China: an interrupted time series analysis

open access: yesBMJ Open, 2023
Objective To evaluate the impacts of the 2017 adjustment of National Reimbursement Drug List (NRDL) on orphan drugs hospital procurement volumes and spending in China.Design We used an interrupted time series design covering the period from 2016 to 2018 ...
Yu Xie   +6 more
doaj   +1 more source

From Mechanoelectric Conversion to Tissue Regeneration: Translational Progress in Piezoelectric Materials

open access: yesAdvanced Materials, EarlyView.
This review highlights recent progress in piezoelectric materials for regenerative medicine, emphasizing their ability to convert mechanical stimuli into bioelectric signals that promote tissue repair. Key discussions cover the intrinsic piezoelectric properties of biological tissues, co‐stimulation cellular mechanisms for tissue regeneration, and ...
Xinyu Wang   +3 more
wiley   +1 more source

G protein-coupled receptor 35: an emerging target in inflammatory and cardiovascular disease [PDF]

open access: yes, 2015
G protein-coupled receptor 35 (GPR35) is an orphan receptor, discovered in 1998, that has garnered interest as a potential therapeutic target through its association with a range of diseases.
Amanda E Mackenzie   +4 more
core   +3 more sources

PTf‐SRiApt Targeting SCAF4‐POLR2A Interaction Suppresses Tumor Growth and Promotes Antitumor Immunity in Triple‐Negative Breast Cancer

open access: yesAdvanced Science, EarlyView.
A phosphorothioate‐modified aptamer PTf‐SRiApt capable of disrupting “undruggable” transcription factor SCAF4‐POLR2A interaction with high inhibitory activity and biological stability is presented here. Through this inhibition, PTf‐SRiApt successfully arrested cell cycle progression, induced cell death, and elevated immune recruitment in SCAF4‐POLR2A ...
Liyan Fei   +13 more
wiley   +1 more source

Impact of environmental and individual factors on COVID-19 mortality in children and adolescents in Mexico: An observational study

open access: yesThe Lancet Regional Health. Americas, 2022
Summary: Background: During the Covid-19 pandemic, children and adolescents faced poverty, potentially dying from preventable causes, or missing out essential vaccines.
Carlos Sanchez-Piedra   +3 more
doaj  

Unraveling the functional role of the orphan solute carrier, SLC22A24 in the transport of steroid conjugates through metabolomic and genome-wide association studies. [PDF]

open access: yes, 2019
Variation in steroid hormone levels has wide implications for health and disease. The genes encoding the proteins involved in steroid disposition represent key determinants of interindividual variation in steroid levels and ultimately, their effects ...
Boerwinkle, Eric   +13 more
core   +2 more sources

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