Results 61 to 70 of about 1,093 (174)

Supply and Demand in the Mathematics of Rare Disease Drug Development: Why Choosing the Right Model Is Crucial

Clinical and Translational Science, Volume 19, Issue 5, May 2026.
ABSTRACT Clinical trials for rare diseases face a fundamental mathematical challenge that conventional randomized controlled trial (RCT) designs cannot overcome. With approximately 95% of the estimated 10,000–16,000 rare diseases lacking approved therapies, and drug development programs failing at rates exceeding 75% in non‐oncology indications, the ...
Marshall L. Summar, Janet Woodcock
wiley   +1 more source

Monoaminergic Networks of Cognitive and Behavioral Symptoms in Early Parkinson's Disease

Movement Disorders, Volume 41, Issue 4, Page 1004-1015, April 2026.
Abstract Background Parkinson's disease (PD) is associated with several behavioral and cognitive symptoms, the neurobiological background of which is not yet fully understood. Objectives The aim was to investigate the association between monoamine function and four specific nonmotor symptoms in early PD using the Parkinson's Progression Markers ...
Kalle J. Niemi, Valtteri Kaasinen, Rimona S. Weil, Juho Joutsa   +3 more
wiley   +1 more source

Is Pitolisant Effective in Reducing Excessive Daytime Sleepiness and Cataplexy in Adults with Narcolepsy? [PDF]

, 2019
Objective: The objective of this selective EBM review is to determine whether or not pitolisant is effective in reducing excessive daytime sleepiness and cataplexy in adults with narcolepsy.
Michael, Christina M
core   +1 more source

Pentylenetetrazol-induced seizures and brain neuroinflammation markers under conditions of pitolisant and pioglitazone use

Journal of Education, Health and Sport
The purpose of the study was to investigate the dynamics of seizure activity and markers of neuroinflammation in kindled rats under conditions of individual and combined use of pitolisant and pioglitazone.
O. Yehorenko, M. Pervak
doaj   +1 more source

Sex differences in neuromodulatory subcortical systems and their implications for Alzheimer's disease

Alzheimer's &Dementia, Volume 22, Issue 3, March 2026.
Abstract Neuromodulatory subcortical systems (NSSs) are uniquely susceptible to dementia‐related pathology, leading to frequent molecular and behavioral impairments associated with altered function of these nuclei. Some of these systems display clear sex‐specific cytoarchitecture and signaling leading to distinct physiology and behavioral outputs in ...
Rosaria J. Rae, Jessica Marie Hunter Alberhasky, Marion Baillet, Debra A. Bangasser, Michael E. Belloy, Anne S. Berry, Chiara Berteotti, Hannah Bow, Rachel Buckley, Jessica Z. K. Caldwell, Matteo Carpi, Benjamin J. Clark, Claire J. Ciampa, Alexander C. Conley, Martin J. Dahl, Zoe R. Donaldson, Alexander J. Ehrenberg, Gillian Einstein, Neus Falgàs, Haley A. Fenlon, Megan C. Fitzhugh, Robert C. Froemke, Clara Gallay, Lea Tenenholz Grinberg, Derek A. Hamilton, Zia Hasan, Oihane Uriarte Huarte, Shaista Jabeen, Heidi I. L. Jacobs, Louis John Kolling, Elouise A. Koops, Sabrina Lenzoni, Claudio Liguori, Riccardo Manca, Catherine A. Marcinkiewcz, Tamunotonye Omoluabi, Rademene Oria, Caitlin A. Orsini, Nancy Elizabeth Ortega, Judy Pa, Nathan S. Pentkowski, Joana B. Pereira, Rhudovic Ramos, Derya Sargin, Abhijit Satpati, Maria Clara Selles, Mabel Seto, Shabana M. Shaik, Shireen Sindi, Gowoon Son, Valentine Ucheagwu, Maxime Van Egroo, Qi Yuan, Michael A. Kelberman   +53 more
wiley   +1 more source

Pitolisant versus placebo or modafinil in patients with narcolepsy: A double-blind, randomised trial

, 2013
Background: Narcolepsy is characterised by excessive daytime sleepiness (EDS) and cataplexy. Histamine neurons are crucial to maintain wakefulness. We assessed the safety and efficacy of pitolisant (previously called BF2.649), a selective histamine H3 ...
Bassetti, Claudio, Mayer, G., HARMONY I Study Grp, Dauvilliers, Yves, Bassetti, C., Lecomte, Jeanne-Marie, Lehert, Philippe, Amulf, I., Rodenbeck, Andrea, Schwartz, J.C., Dauvilliers, Y., Ding, C.L., Lecomte, J.M., Lammers, Gert Jan, Mayer, Geert, Lammers, G.J., Lehert, P., Arnulf, Isabelle, Schwartz, Jean-Charles, Rodenbeck, A., Ding, Claire-Li   +20 more
core   +1 more source

Nonclinical cardiovascular safety of pitolisant: comparing International Conference on Harmonization S7B and Comprehensive in vitro Pro-arrhythmia Assay initiative studies [PDF]

, 2017
Background and purpose: We evaluated the concordance of results from two sets of nonclinical cardiovascular safety studies on pitolisant. Experimental approach: Nonclinical studies envisaged both in the ICH S7B guideline and Comprehensive in vitro ...
Laurent Landais, Ligneau, Xavier, Mirams, Gary, Philippe Robert, Jean-Charles Schwartz, Lecomte, Jeanne-Marie, Shah, Rashmi, Jeanne-Marie Lecomte, Faivre, Jean-Fran�ois, Xavier Ligneau, Berrebi-Bertrand, Isabelle, Landais, Laurent, Shah, Rashmi R., Mirams, Gary R., Gary R Mirams, Maison-Blanche, Pierre, Faivre, Jean-François, Pierre Maison-Blanche, Isabelle Berrebi-Bertrand, Robert, Philippe, Schwartz, Jean-Charles, Jean-François Faivre, Rashmi R Shah   +22 more
core   +1 more source

Activity‐restoring mutations in the histamine H3 receptor increase constitutive activity and reduce structural stability

Protein Science, Volume 35, Issue 1, January 2026.
Abstract The histamine H3 receptor (H3R) is a class A G protein‐coupled receptor (GPCR) that regulates neurotransmitter release in the central nervous system. Although structures of H3R in both inactive and active states have been elucidated, the functional roles of specific residues remain unclear.
Ami Nakajima, Hiroto Kaneko, Kosuke Oyama, Misumi Kuchiji, Ayane Itakura, Chiaki Arai, Mitsunori Shiroishi   +6 more
wiley   +1 more source

Time to Onset of Response to Pitolisant for the Treatment of Excessive Daytime Sleepiness and Cataplexy in Patients With Narcolepsy: An Analysis of Randomized, Placebo-Controlled Trials

, 2021
International audienceBackground: Pitolisant is approved in the USA and Europe for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy.Objective: Analyses evaluated the time to onset of clinical response during treatment ...
Watson, Nathaniel, Dauvilliers, Yves, Davis, Craig, Vaughn, Ben, Dayno, Jeffrey, Schwartz, Jean-Charles, Zarycranski, Donna   +6 more
core   +1 more source

Living with Narcolepsy: Current Management Strategies, Future Prospects, and Overlooked Real-Life Concerns

Nature and Science of Sleep, 2020
Emily C Barker,1 Julie Flygare,2 Shalini Paruthi,3,4 Katherine M Sharkey5– 7 1Case Western Reserve University School of Medicine, Department of Pediatrics, Cleveland, OH, USA; 2Project Sleep, Los Angeles, CA, USA; 3Sleep Medicine and Research ...
Barker EC, Flygare J, Paruthi S, Sharkey KM   +3 more
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