Results 41 to 50 of about 159,613 (271)

The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases

open access: yesOrphanet Journal of Rare Diseases, 2011
Background Over 95% of rare diseases lack treatments despite many successful treatment studies in animal models. To improve access to treatments, the Accelerated Approval (AA) regulations were implemented allowing the use of surrogate endpoints to ...
Kakkis Emil D, Miyamoto Brigitta E
doaj   +1 more source

The anti‐CRISPR protein AcrIE8.1 inhibits the type I‐E CRISPR‐Cas system by directly binding to the Cascade subunit Cas11

open access: yesFEBS Letters, EarlyView.
In this study, we present the structure of AcrIE8.1, a previously uncharacterized anti‐CRISPR protein that inhibits the type I‐E CRISPR‐Cas system. Through a combination of structural and biochemical analyses, we demonstrate that AcrIE8.1 directly binds to the Cas11 subunit of the Cascade complex to inhibit the CRISPR‐Cas system.
Young Woo Kang, Hyun Ho Park
wiley   +1 more source

Cancer Drugs Approved Based on Surrogate Endpoint: A Retrospective Observational Study in the United States and China

open access: yesCancer Medicine
Background Hundreds of cancer drugs are approved globally based on surrogate endpoint response rate (RR). However, the characteristics of RR‐based approvals remain unknown.
Ting Zhu, Jinjia Zhong, Yafang Huang
doaj   +1 more source

The role of histone modifications in transcription regulation upon DNA damage

open access: yesFEBS Letters, EarlyView.
This review discusses the critical role of histone modifications in regulating gene expression during the DNA damage response (DDR). By modulating chromatin structure and recruiting repair factors, these post‐translational modifications fine‐tune transcriptional programmes to maintain genomic stability.
Angelina Job Kolady, Siyao Wang
wiley   +1 more source

PI3k Inhibitors in NHL and CLL: An Unfulfilled Promise

open access: yesBlood and Lymphatic Cancer: Targets and Therapy, 2023
Naji Bou Zeid,1 Victor Yazbeck2 1Hôtel-Dieu de France Hospital, Saint Joseph University of Beirut, Beirut, Lebanon; 2Massey Cancer Center, Virginia Commonwealth University, Richmond, VA, USACorrespondence: Victor Yazbeck, Virginia Commonwealth University,
Bou Zeid N, Yazbeck V
doaj  

Time to inclusion in clinical guidance documents for non-oncological orphan drugs and biologics with expedited FDA designations: a retrospective survival analysis

open access: yesBMJ Open, 2021
Objectives Drug and biological products that treat rare, serious or life-threatening conditions can receive US Food and Drug Administration (FDA) orphan designation and expedited programme designations (accelerated approval, breakthrough therapy, fast ...
Dima M Qato   +3 more
doaj   +1 more source

Time after time – circadian clocks through the lens of oscillator theory

open access: yesFEBS Letters, EarlyView.
Oscillator theory bridges physics and circadian biology. Damped oscillators require external drivers, while limit cycles emerge from delayed feedback and nonlinearities. Coupling enables tissue‐level coherence, and entrainment aligns internal clocks with environmental cues.
Marta del Olmo   +2 more
wiley   +1 more source

Biomarkers for immunotherapy in bladder cancer: a moving target

open access: yesJournal for ImmunoTherapy of Cancer, 2017
Treatment options for metastatic urothelial carcinoma (mUC) remained relative unchanged over the last 30 years with combination chemotherapy as the mainstay of treatment.
David H. Aggen, Charles G. Drake
doaj   +1 more source

An upstream open reading frame regulates expression of the mitochondrial protein Slm35 and mitophagy flux

open access: yesFEBS Letters, EarlyView.
This study reveals how the mitochondrial protein Slm35 is regulated in Saccharomyces cerevisiae. The authors identify stress‐responsive DNA elements and two upstream open reading frames (uORFs) in the 5′ untranslated region of SLM35. One uORF restricts translation, and its mutation increases Slm35 protein levels and mitophagy.
Hernán Romo‐Casanueva   +5 more
wiley   +1 more source

Trends in FDA drug approvals over last 2 decades: An observational study

open access: yesJournal of Family Medicine and Primary Care, 2020
Introduction: The discovery of novel drugs is critical for pharmaceutical research and development as well as for patient treatment. Repurposing existing drugs that may have anticipated effects as potential candidate is one way to meet this important ...
Angelika Batta   +2 more
doaj   +1 more source

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