Results 91 to 100 of about 32,298 (248)

Cardiac MIBG Scintigraphy in Neurodegenerative Parkinsonism: Limitations in Clinical Practice

Movement Disorders Clinical Practice, EarlyView.
Abstract Background Reduced cardiac uptake on 123Iodine‐metaiodobenzylguanidine (MIBG) scintigraphy is a valuable tool for differentiating neurodegenerative parkinsonism but interpretation can be difficult due to comorbidities and drug‐tracer interactions.
Frank Jagusch, Elena Redl, Christoph Theyer, Atbin Djamshidian, Alessandra Fanciulli, Werner Poewe, Philipp Mahlknecht, Beatrice Heim, Klaus Seppi, Florian Krismer   +9 more
wiley   +1 more source

Clinical development and marketing application review times for novel orphan-designated drugs

Frontiers in Medicine
Development of an orphan-designated drug has been more challenging and financially less attractive than that of other drugs due to low prevalence of the condition, poorly defined biomarkers and lack of experience of healthcare providers in diagnosing and
Ebru Demirci, Jennifer Knicley, Lori Fiorentino   +2 more
doaj   +1 more source

Orphan drugs [PDF]

Nature, 1992
openaire   +2 more sources

Systematic Review of the Huntington's Disease Drug Development Pipeline, 2014 to 2025

Movement Disorders, EarlyView.
Abstract Background In the past decade, significant advances have improved our understanding of the mechanisms underlying HD pathobiology leading to several putative therapeutic targets for HD. Objective The aim was to describe the Huntington's disease (HD) drug development clinical pipeline.
Pavlina Konstantinova, Jenny Townhill, Juliana Bronzova, Viktoria Andreeva, Swati Sathe, Cristina Sampaio   +5 more
wiley   +1 more source

Small‐Molecule Kinase Inhibitors Modulating Circadian Rhythms

Medicinal Research Reviews, EarlyView.
ABSTRACT The circadian clock mechanism generates 24‐h rhythms crucial for regulating various physiological processes, and its dysregulation has been implicated in numerous diseases. In cells, the circadian clock operates through a transcriptional‐translational feedback loop, where phosphorylation plays a pivotal role in maintaining accurate circadian ...
Irene Castellino, Manuela Bartolini, Rudolf Andrys, Kamil Musilek   +3 more
wiley   +1 more source

Late INa as a Therapeutic Target: New Strategies, Computational Modelling, Drug Development, and Clinical Translation

Medicinal Research Reviews, EarlyView.
ABSTRACT The Nav1.5 channel, a major isoform of voltage‐gated sodium ion channel, is mainly found in ventricular cardiomyocytes, playing a key role in generating essential cardiac action potentials for normal heart rhythms. Mutations in Nav1.5 have been associated with severe heart conditions such as long QT syndrome, Brugada syndrome, cardiac ...
Arkapravo Chattopadhyay, Lynn C. Lunsonga, Peter E. Light, Khaled Barakat   +3 more
wiley   +1 more source

Clinical benefit, price and epidemiology of orphan drugs approved in China: a cross-sectional analysis of orphan drug policy (ODP) in China

BMJ Open
Objectives To evaluate the regulatory approval, epidemiology, clinical benefits and pricing of orphan drugs approved by the National Medical Products Administration (NMPA) in China.Design Cross-sectional analysis.Study sample Orphan drugs approved by the
Bin Jiang, Minghui Li, Jing Yuan, Z Kevin Lu, Xiangxiang Jiang   +4 more
doaj   +1 more source

Analysis of marketed orphan drugs in China. [PDF]

Intractable Rare Dis Res, 2023
Zhi W, Liu M, Yang D, Zhang S, Lu Y, Han J.   +5 more
europepmc   +1 more source

Artificial Intelligence‐Driven Natural Product Drug Discovery: From Computational Genome Mining to Clinical Translation

Medicinal Research Reviews, EarlyView.
ABSTRACT Natural products (NPs) have historically yielded numerous therapeutic agents, yet their integration into modern drug discovery has been constrained by chemical complexity, low abundance, laborious dereplication, and limited target annotation.
Antonio Lavecchia
wiley   +1 more source

Utilization and affordability of health insurance coverage for rare disease drugs in a first-tier city in Northeast China from 2018 to 2021: a study based on the health insurance claims database

International Journal for Equity in Health
Objective The accessibility issue of orphan drugs in China is prominent. Based on real-world data from a tier-one city in Northeast China, this study aims to analyze the current usage and affordability of orphan drugs for rare diseases.
Yaqun Wang, Na Zhou, Baoxin Li, Zixuan Lv, Shengnan Duan, Xin Li, Ni Yuan   +6 more
doaj   +1 more source