Results 71 to 80 of about 3,868,024 (353)

Progastrin Promotes Colorectal Cancer Stem Cell‐Like Properties via the Receptor PZR

open access: yesAdvanced Science, EarlyView.
The tumor microenvironment supports colorectal cancer (CRC) progression by maintaining cancer stem cells (CSCs) through progastrin (PG). This study identifies Protein Zero‐Related protein (PZR) as an essential receptor for PG activity, promoting CSC‐like signaling via SHP2/SRC/β‐catenin.
Julie Nguyen   +22 more
wiley   +1 more source

Analysis of the first ten years of FDA’s rare pediatric disease priority review voucher program: designations, diseases, and drug development

open access: yesOrphanet Journal of Rare Diseases
Background The Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) Program was enacted in 2012 to support the development of new products for children.
Catherine Mease   +5 more
doaj   +1 more source

Sleep Deprivation Activates a Conserved Lactate‐H3K18la‐RORα Axis Driving Neutrophilic Inflammation Across Species

open access: yesAdvanced Science, EarlyView.
Sleep deprivation triggers systemic immune activation characterized by neutrophil accumulation and cytokine release. This study reveals a conserved metabolic‒epigenetic mechanism whereby lactate‐induced H3K18 lactylation upregulates RORα expression, promoting neutrophilic inflammation.
Ren Zhou   +15 more
wiley   +1 more source

The Promising Viral Threat to Bacterial Resistance: The Uncertain Patentability of Phage Therapeutics and the Necessity of Alternative Incentives [PDF]

open access: yes, 2019
Bacteriophages, or “phages,” are a category of highly adept and adaptable viruses that can infect and kill bacteria. With concerns over the burgeoning antibiotic-resistance crisis looming in recent years, scientists and policymakers have expressed a ...
Todd, Kelly
core   +2 more sources

YTHDC1 Is Essential for Postnatal Liver Development and Homeostasis

open access: yesAdvanced Science, EarlyView.
This study identifies YTHDC1 as a key regulator of postnatal liver development and disease. Hepatocyte‐specific deletion of Ythdc1 impairs hepatocyte maturation, causing liver injury, contributing to nonalcoholic steatohepatitis and hepatocellular carcinoma.
Xinzhi Li   +8 more
wiley   +1 more source

Disease awareness or subtle product placement? Orphan diseases featured in the television series “House, M.D.” - a cross-sectional analysis

open access: yesBMC Medical Ethics, 2020
Background Approximately 7% of the general population is affected by an orphan disease, which, in the United States, is defined as affecting fewer than 1 in 1500 people. Disease awareness is often low and time-to-diagnosis delayed. Different legislations
Konstantin Mechler   +3 more
doaj   +1 more source

Expert Consensus on Clobazam in the Treatment of Refractory Epilepsy (2022)

open access: yesXiehe Yixue Zazhi, 2022
After regular anti-epileptic drug treatment, the symptoms of most patients with epilepsy can be well controlled or relieved, but 30%-40% of patients with epilepsy, after long-term drug treatment, still suffer from repeated seizures and develop refractory
Multi-disciplinary Team for Rare Diseases, Peking Union Medical College Hospital National Rare Diseases Committee
doaj   +1 more source

Drugs for exceptionally rare diseases: a commentary on Hughes et al [PDF]

open access: yes, 2006
Recently in this journal, Hughes and colleagues discussed special funding status to ultra-orphan drugs. They concluded that there should be a uniform policy for the provision of orphan drugs across Europe; that complete restriction was impractical, and ...
Claxton, K.   +3 more
core  

Repurposing With Purpose: Treatment of Bachmann–Bupp Syndrome With Eflornithine and Implications for Other Polyaminopathies

open access: yesAmerican Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.
ABSTRACT Rare diseases impact approximately 1 in 10 people worldwide, and yet, less than 5% of all rare diseases currently have an approved treatment option available. This is due to many challenges unique to rare diseases, including small, diverse patient populations, the cost of drug development that is not proportionate to the number of patients who
Caleb P. Bupp   +7 more
wiley   +1 more source

Autosomal Recessive Cerebellar Ataxias: Translating Genes to Therapies

open access: yesAnnals of Neurology, EarlyView.
Autosomal recessive cerebellar ataxias are disabling neurodegenerative genetic conditions affecting balance and coordination. Advancements in genomic testing have improved diagnosis, leading to a new focus on the development of targeted precision therapeutics addressing cellular, biochemical, and genetic disease mechanisms with a resulting emphasis on ...
Brent L. Fogel   +10 more
wiley   +1 more source

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