Results 41 to 50 of about 22,884 (253)

Venture philanthropy in rare disease therapy

Rare
Purpose: Rare diseases affect ∼350 million people globally, yet 94 % of those affected lack effective treatments due to funding gaps and high R&D costs.
JA Levine
doaj   +1 more source

The work, goals, challenges, achievements, and recommendations of orphan medicinal product organizations in India: an interview-based study

Orphanet Journal of Rare Diseases, 2019
Background Orphan medicinal products (OMPs) are intended for the diagnosis, prevention, management or treatment of rare diseases (RDs). Each RD affects only a small fraction of the population, and therefore, historically, industry hesitated to undertake ...
Mohua Chakraborty Choudhury, Gayatri Saberwal   +1 more
doaj   +1 more source

Tetrahedral DNA Nanostructure‐Based Biomimetic Nanovesicles Attenuate Sepsis‐Associated ARDS by Suppressing Glycolysis via the BMAL1/PFKFB3 Axis

Advanced Science, EarlyView.
Upon inhalation, RM@TNT could persist long‐term in the diseased lungs, while undergoing disintegration to release TNT specifically within the ROS‐rich pathological microenvironments of SA‐ARDS. The released TNT was then precisely delivered to AMs via Tuftsin, where it released Nob intracellularly to activate BMAL1 expression, thus inhibiting AM ...
Yunlong Zhang, Bin Li, Zhijin Fan, Yan Yan, Fei Ma, Changting He, Shiping Liu, Mingliang Pan, Zhou Pan, Huijuan Wang, Xinting Fu, Jiamei Wang, Yue Jia, Qin Gu, Duo Jiang, Xueting Liu, Bohua Ren, Qinqin Wang, Yuehua Hei, Han Duan, Yanqiu Wu, Zihui Wei, Liying Zhan, Yuhui Liao   +23 more
wiley   +1 more source

Decoding the Mechanism of Action of a Parasite TGFβ Antagonist Inspires the Creation of Cell‐Type‐Specific TGFβ Modulators

Advanced Science, EarlyView.
The mammalian TGFβ interacts with ubiquitously expressed TGFBR1 and TGFBR2, and current TGFβ‐targeting agents are non‐cell‐selective. The cooperative interaction of the modular parasite TGFβ antagonist with multiple host (co‐)receptors empowers the design of TGM chimeras and bispecific antibodies that activate or inhibit TGFβ signaling in a cell ...
Maarten van Dinther, Tristin Schwartze, Jiying Zhang, Kun Fan, Gerard van der Zon, Luke Power, Cynthia S. Hinck, Claire Ciancia, Ananya Mukundan, Roman Gonzalez‐Prieto, Peter van Veelen, Rick M. Maizels, Andrew P. Hinck, Peter ten Dijke   +13 more
wiley   +1 more source

New Approaches and Strategies for the Repurposing of Iron Chelating/Antioxidant Drugs for Diseases of Free Radical Pathology in Medicine

Antioxidants
There is an urgent need for new approaches and strategies for the introduction of antioxidant drugs in medicine. Despite hundreds of clinical trials with potential antioxidants, no antioxidant drugs have so far been developed for clinical use; this is ...
George J. Kontoghiorghes
doaj   +1 more source

Toward Predictable Nanomedicine: Current Forecasting Frameworks for Nanoparticle–Biology Interactions

Advanced Intelligent Discovery, EarlyView.
Predictive models successfully screen nanoparticles for toxicity and cellular uptake. Yet, complex biological dynamics and sparse, nonstandardized data limit their accuracy. The field urgently needs integrated artificial intelligence/machine learning, systems biology, and open‐access data protocols to bridge the gap between materials science and safe ...
Mariya L. Ivanova, Michael Nicholls, Nicola Russo, Gueorgui Mihaylov, Konstantin Nikolic   +4 more
wiley   +1 more source

Failures to further developing orphan medicinal products after designation granted in Europe: an analysis of marketing authorisation failures and abandoned drugs [PDF]

BMJ Open, 2017
Objectives The research and development process in the field of rare diseases is characterised by many well-known difficulties, and a large percentage of orphan medicinal products do not reach the marketing approval.
Giannuzzi, Viviana, Landi, Annalisa, Bosone, Enrico, Giannuzzi, Floriana, Nicotri, Stefano, Torrent-Farnell, Josep, Bonifazi, Fedele, Felisi, Mariagrazia, Bonifazi, Donato, Ceci, Adriana   +9 more
openaire   +2 more sources

Facilitating Expedited Drug Approval—the Past, Present, and Future of Clinical Trials for Pediatric Rheumatic Disease

Arthritis Care &Research, EarlyView.
Pamela F. Weiss, Hermine I. Brunner
wiley   +1 more source

From cramming to binge‐watching: Integrating documentary‐based assessment into a pharmacology and toxicology curriculum—a qualitative study

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aims In the context of pharmacology and toxicology education, there is a growing shift toward programmatic assessment models that prioritize longitudinal learning, reflection and development of higher‐order cognitive skills. As part of this transition, we are exploring alternative and more meaningful forms of assessment. This qualitative study
Narin Akrawi, Heleen van der Sijs, Floor van Rosse   +2 more
wiley   +1 more source

The impact of China’s drug regulatory reform on access to orphan drugs: a cross-sectional study of diseases listed in the rare disease catalog

Frontiers in Pharmacology
ObjectiveChina released two batches of the Rare Disease Catalog in 2018 and 2023, respectively, listing a total of 207 diseases. This study aims to analyze the accessibility of medications for diseases included in the catalog and assess the impact of ...
Yipeng Lan, Xiaofeng Lin, Zhe Huang, Zhe Huang, Wang Zan   +4 more
doaj   +1 more source