Results 161 to 170 of about 22,949 (304)

A Systematic Review on Disease‐Modifying Therapies in Parkinsonian Disorders

Clinical Pharmacology &Therapeutics, EarlyView.
Parkinsonian disorders, including Parkinson's disease, Lewy body dementia, multiple system atrophy, and progressive supranuclear palsy, are progressive neurodegenerative conditions with no treatment options to slow disease progression. This systematic review provides an overview of evidence of disease‐modifying therapies that have been evaluated in ...
Pepijn P.N.M. Eijsvogel, Lars M.G. Smits, Philip H.C. Kremer, Geert Jan Groeneveld   +3 more
wiley   +1 more source

Transcranial Magnetic Stimulation as a Translational Biomarker in Early‐Phase Anti‐Seizure Medication Development: A Randomized, Double‐Blind, Placebo‐Controlled Study in Generalized Epilepsy

Clinical Pharmacology &Therapeutics, EarlyView.
One‐third of epilepsy patients remain treatment‐resistant, underscoring the need for novel anti‐seizure medications (ASMs) and reliable biomarkers of central target engagement. Cortical hyperexcitability is a hallmark of epilepsy, making excitability a valuable pharmacodynamic biomarker for early‐phase drug development supporting go/no‐go decision ...
Catherine M. E. de Cuba, Titia Q. Ruijs, Annika A. de Goede, Robert J. Doll, Maria Juachon, Michel J. A. M. van Putten, Geert Jan Groeneveld, Jules A. A. C. Heuberger   +7 more
wiley   +1 more source

Considerations for drug trials in hypertrophic cardiomyopathy

ESC Heart Failure, Volume 12, Issue 2, Page 1095-1112, April 2025.
Abstract Hypertrophic cardiomyopathy (HCM) is a heterogeneous condition with potentially serious manifestations. Management has traditionally comprised therapies to palliate symptoms and implantable cardioverter‐defibrillators to prevent sudden cardiac death. The need for disease‐modifying therapies has been recognized for decades.
John P. Farrant, Matthias Schmitt, Anna B. Reid, Clifford J. Garratt, William G. Newman, Aneil Malhotra, Rhys Beynon, Masliza Mahmod, Betty Raman, Robert M. Cooper, Dana Dawson, Thomas Green, Sanjay K. Prasad, Anvesha Singh, Susanna Dodd, Hugh Watkins, Stefan Neubauer, Christopher A. Miller   +17 more
wiley   +1 more source

Effectiveness and safety of cenobamate after conversion to monotherapy or dual therapy in real‐world clinical practice

Epileptic Disorders, EarlyView.
Abstract Objective Patients with drug‐resistant epilepsy (DRE) typically take multiple anti‐seizure medications (ASMs) and are at risk of treatment‐related adverse events (AEs). This study assessed the impact of cenobamate monotherapy or dual therapy in patients with drug‐resistant epilepsy.
Álvaro Sánchez‐Guijo Benavente, Guillermina García Martín, Estuardo Daniel Castro Ruiz, Pablo Cabezudo García, Yolanda López Moreno, José Carlos Estévez María, Irene Lorite Fuentes, Sergio Navarro Conti, Raquel Calle Calle, Alejandro Fernández Cabrera, Isabel Ros González, Dulce Campos Blanco, María Isabel Chamorro Muñoz, Pedro Jesús Serrano Castro   +13 more
wiley   +1 more source

Allosteric Modulators of Drug Targets [PDF]

Journal of Medicinal Chemistry, 2018
openaire   +2 more sources

Stiripentol: Unpublished results from the first phase 2 clinical trial in Lennox–Gastaut syndrome conducted in the early 1990s

Epilepsia, EarlyView.
Abstract Objective This study was undertaken to present the results of an exploratory phase 2 trial of stiripentol in Lennox–Gastaut syndrome (LGS). Methods This exploratory single‐blind, single‐arm, nonrandomized sequential‐period phase 2 study was conducted at four centers in France between January 1989 and August 1993.
Stéphane Auvin, Benjamin Serraz, Jérémie Lespinasse, Laurent Chancharme   +3 more
wiley   +1 more source

Positive allosteric modulator selective for adult muscle nicotinic acetylcholine receptor. [PDF]

Proc Natl Acad Sci U S A
Webster RG, Alabaf S, Li A, Beerli R, Siehler S, Imbert PE, Lee L, Cossins J, Koziczak-Holbro M, Flotte L, Gaudet L, Gerwin N, Beeson D, Dong YY.   +13 more
europepmc   +1 more source

Soticlestat as an adjunctive therapy in children and young adults with Dravet syndrome

Epilepsia, EarlyView.
Overview of the phase 3 trial evaluating soticlestat as adjunctive therapy in children and young adults with Dravet syndrome. Abstract Objective This study evaluated the efficacy, safety, and tolerability of soticlestat as adjunctive therapy in children and young adults with Dravet syndrome (DS).
Joseph Sullivan, Kette Valente, Vicente Villanueva, Adam Strzelczyk, Rima Nabbout, Eiji Nakagawa, Yuehua Zhang, Marta Zolnowska, Yasir Khan, Cheng Dong, Samuel Hsiao, Sarah I. Sheikh, Philipp von Rosenstiel, Mahnaz Asgharnejad, Venkatesha Murthy   +14 more
wiley   +1 more source

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

Optical control of pain in vivo with a photoactive mGlu5 receptor negative allosteric modulator

Light-operated drugs constitute a major target in drug discovery, since they may provide spatiotemporal resolution for the treatment of complex diseases (i.e. chronic pain). JF-NP-26 is an inactive photocaged derivative of the metabotropic glutamate type
Bresolí-Obach, Roger, Font, Joan, López-Cano, Marc, Llebaria Soldevila, Amadeu, Ciruela Alférez, Francisco, Scarselli, Pamela, Goudet, Cyril, Nonell, S. (Santi), Catena, Juanlo, Malhaire, Fanny, Di Pietro, Paola, Battaglia, Giuseppe, Giraldo, Jesús, Pin, Jean-Philippe, Nicoletti, Ferdinando, Rovira, Xavier, Fernández Dueñas, Víctor, Notartomaso, Serena   +17 more
core   +1 more source