Results 71 to 80 of about 22,884 (253)

Precision therapies for genetic epilepsies in 2025: Promises and pitfalls

Epilepsia Open, EarlyView.
Abstract By targeting the underlying etiology, precision therapies offer an exciting paradigm shift to improve the stagnant outcomes of drug‐resistant epilepsies, including developmental and epileptic encephalopathies. Unlike conventional antiseizure medications (ASMs) which only treat the symptoms (seizures) but have no effect on the underlying ...
Shuyu Wang, Emilio Perucca, Samuel F. Berkovic, Piero Perucca   +3 more
wiley   +1 more source

Orphan receptor GPR176 in hepatic stellate cells exerts a profibrotic role in chronic liver disease

JHEP Reports
Background & Aims: Chronic liver disease (CLD) remains a global health issue associated with a significant disease burden. Liver fibrosis, a hallmark of CLD, is characterised by the activation of hepatic stellate cells (HSCs) that gain profibrotic ...
Vincent De Smet, Elif Gürbüz, Nathalie Eysackers, Liza Dewyse, Ayla Smout, Mina Kazemzadeh Dastjerd, Pierre Lefesvre, Nouredin Messaoudi, Hendrik Reynaert, Stefaan Verhulst, Inge Mannaerts, Leo A. van Grunsven   +11 more
doaj   +1 more source

Neurolathyrism in Sub‐Saharan Africa—Assessing the Neurotoxic Risks of Lathyrus sativus Amid Drought and Food Security Challenges

Food Safety and Health, EarlyView.
Representation of grass pea consumption in drought‐stricken sub‐Saharan Africa sustains nutrition, but excess β‐ODAP exposure due to multiple reasons triggers neurolathyrism, a progressive neurotoxic disorder. ABSTRACT Neurolathyrism is a progressive motor neuron disease due to the consumption of Lathyrus sativus (grass pea) over long periods.
Biruk Demisse Ayalew, Muhammad Umar, Pakeezah Tabasum, Maria Qadri, Talha Farooq, Eskeatnaf Yosef Getachew, Zemichael Getu Alemayehu, Eden Haile Hagos, Hailemariam Shimelis Gebeyehu, Hanna Tsehay Abebe, Nuradin Abdi Ali, David Chinaecherem Innocent, Temesgen Mamo Sharew   +12 more
wiley   +1 more source

Access to Orphan Medicinal Products in Bulgaria: An Analysis of the Positive Drug List and Individual Access Schemes

Healthcare
Background/Objectives: Orphan medicinal products offer essential treatments for rare diseases, but patient access varies across European Union countries despite a common regulatory framework. In Bulgaria, access is primarily through inclusion in the positive drug list following health technology assessment or via individual access schemes under ...
Rumen Stefanov, Ralitsa Raycheva, Kostadin Kostadinov, Georgi Stefanov, Iva Zdravkova-Aneva, Elizabet Dzhambazova, Georgi Iskrov   +6 more
openaire   +3 more sources

Recurrent Respiratory Papillomatosis Foundation Position Statement on the Management of Adults With RRP

The Laryngoscope, EarlyView.
ABSTRACT Objective With regulatory approval of HPV‐specific immunotherapy for recurrent respiratory papillomatosis (RRP) and growing experience with systemic bevacizumab, a management algorithm incorporating these medical treatments is warranted. Data Sources and Methods RRP Foundation (RRPF) Key Opinion Leaders offer a proposed management algorithm ...
Simon R. Best, Aaron D. Friedman, Clark A. Rosen, Robert T. Sataloff, Laura A. Matrka, H. Steven Sims, David E. Rosow, Nabil F. Saba, David G. Lott, Adam M. Klein, Ted Mau, Milan R. Amin, Kathryn A. Wikenheiser‐Brokamp, Scott M. Norberg, Kim McClellan, Geoffrey D. Young, Clint T. Allen, on behalf of the Recurrent Respiratory Papillomatosis Foundation   +17 more
wiley   +1 more source

Drivers of Pharmaceutical Compounding: Regional Experience Analysis Using Irkutsk Pharmacy Organisations as a Case Study

Регуляторные исследования и экспертиза лекарственных средств
INTRODUCTION. National pharmacy compounding is a priority for providing the population with medicinal products, particularly the medicinal products lacking in the Russian Federation.
G. N. Kovalskaya, N. V. Verlan, E. N. Mikhalevich   +2 more
doaj   +1 more source

Advances in GPCRs Associated With Wnt Signaling Within the Auditory System

Medicine Bulletin, EarlyView.
ABSTRACT G protein‐coupled receptors (GPCRs) that interact with the Wnt signaling pathway are pivotal for auditory system homeostasis, as they orchestrate inner ear development, hair cell (HC) regeneration, and hearing preservation. Frizzled (FZD) receptors, the core Wnt‐related GPCRs, bind Wnt ligands and co‐receptors (e.g., LRP5/6) to activate both ...
Liang Wang, Changling Xu, Jiangang Fan, Renjie Chai   +3 more
wiley   +1 more source

Considerations for Initiation and Maintenance of Foslevodopa/Foscarbidopa for Advanced Parkinson's Disease

Movement Disorders Clinical Practice, EarlyView.
Abstract Background As Parkinson's disease (PD) progresses, motor fluctuations become increasingly difficult to manage with oral medications. Foslevodopa/foscarbidopa (LDp/CDp), delivered as a continuous 24‐h/day subcutaneous infusion, offers continuous levodopa delivery and stable plasma levodopa levels that reduce motor fluctuations. LDp/CDp has been
K. Ray Chaudhuri, Bruno Bergmans, Eric Freire‐Alvarez, Lucie Hopes, Drew S. Kern, Robert S. Kirsner, Giulia Lazzeri, Pedro Mendes‐Bastos, Noriko Nishikawa, Per Odin, Karolina Poplawska‐Domaszewicz, Rajesh Pahwa, Yuval Ramot, Klaus Seppi, Tobias Warnecke, Robert Wiggins, Megha B. Shah, Pavnit Kukreja, Bjoern Fritz, Koray Onuk, Stuart H. Isaacson   +20 more
wiley   +1 more source

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