Results 161 to 170 of about 104,915 (286)

A phase 3, randomized clinical trial of soticlestat as adjunctive therapy for Lennox–Gastaut syndrome

Epilepsia, EarlyView.
Soticlestat as adjunctive therapy for Lennox–Gastaut syndrome. Abstract Objective There remains a need for new treatments for Lennox–Gastaut syndrome (LGS), a developmental and epileptic encephalopathy with a heterogenous patient population that often requires polytherapy. The phase 3, randomized SKYWAY study (NCT04938427) investigated the efficacy and
Renzo Guerrini, Eric D. Marsh, Wei‐Ping Liao, Katsumi Imai, Ruzica Kravljanac, Anna Altmann, Cecil D. Hahn, Stéphane Auvin, Jimmy Schiemann, Yasir Khan, Pranab Mitra, Sarah I. Sheikh, Philipp von Rosenstiel, Mahnaz Asgharnejad, Dennis Dlugos, Venkatesha Murthy   +15 more
wiley   +1 more source

Inherited metabolic epilepsies–established diseases, new approaches

Epilepsia Open, EarlyView.
Abstract Inherited metabolic epilepsies (IMEs) represent the inherited metabolic disorders (IMDs) in which epilepsy is a prevailing component, often determining other neurodevelopmental outcomes associated with the disorder. The different metabolic pathways affected by individual IMEs are the basis of their rarity and heterogeneity.
Itay Tokatly Latzer, Phillip L. Pearl
wiley   +1 more source

The analysis of specialists’ opinion on the implementation of concept of risk management in clinical trials of drugs

Zaporožskij Medicinskij Žurnal, 2016
At the modern stage of the pharmaceutical industry development quality risk management becomes an integral part of all the processes of drug development and clinical trials (CT).
E. A. Zupanets, V. Ye. Dobrova
doaj  

Circadian rhythms in diabetic retinopathy: an overview of pathogenesis and investigational drugs. [PDF]

Expert Opin Investig Drugs, 2020
Bhatwadekar AD, Rameswara V.
europepmc   +1 more source

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

Ameliorating Seizures in Dravet Syndrome: A Review of Newly Approved and Investigational Drugs, RNA and Gene-Based Therapies. [PDF]

CNS Drugs
Balestrini S, Scheffer IE.
europepmc   +1 more source

Tumor‐educated platelets in cancer diagnostics and prognostics: A critical appraisal and roadmap for clinical translation

International Journal of Cancer, EarlyView.
Abstract Tumor‐educated platelets (TEPs) are emerging as a compelling frontier in liquid biopsy, functioning as dynamic, systemic sensors that sequester and process tumor‐derived biomolecules. This interaction imprints an integrated molecular signature of malignancy—spanning the transcriptome, proteome, lipidome, and crucially, the captured genome ...
Whi‐An Kwon, Min‐Kyung Lee, Eunyong Ahn, Heeyeon Kim, Yong Sang Song, Taejin Ahn   +5 more
wiley   +1 more source

Advances in treatment of hepatitis delta virus infection: Update on novel investigational drugs. [PDF]

World J Virol
Park J, Sayed A, Nasir SA, Lim JK.
europepmc   +1 more source

Real‐world effectiveness and safety with nivolumab plus ipilimumab or nivolumab alone in patients with or without melanoma brain metastasis: Results from the German noninterventional NICO study

International Journal of Cancer, EarlyView.
What's New? Although patients with melanoma brain metastases (MBM) may benefit from treatment with immune checkpoint inhibitors (ICIs), which are known to improve survival in advanced melanoma, MBM patients have often been excluded from trials evaluating ICIs.
Ralf Gutzmer, Michael Weichenthal, Thomas Eigentler, Peter Mohr, Thomas Sickmann, Pia Dücker, Christoffer Gebhardt, Sebastian Haferkamp, Katharina C. Kähler, Friedegund Meier, Claudia Pföhler, Anca Sindrilaru, Patrick Terheyden, Imke von Wasielewski, Selma Ugurel, Jens Ulrich, Jochen Utikal, Carsten Weishaupt, Dirk Schadendorf   +18 more
wiley   +1 more source

Identification, validation, and characterization of approved and investigational drugs interfering with the SARS-CoV-2 endoribonuclease Nsp15. [PDF]

Protein Sci
Chatziefthymiou SD, Kuzikov M, Afandi S, Kovacs G, Srivastava S, Zaliani A, Gruzinov A, Pompidor G, Lunelli M, Ahmed GR, Labahn J, Hakanpää J, Windshügel B, Kolbe M.   +13 more
europepmc   +1 more source