Results 201 to 210 of about 3,868,024 (353)

Drug discovery and preclinical testing of drug candidates for developmental and epileptic encephalopathies

Epilepsia, EarlyView.
Abstract Drug development for developmental and epileptic encephalopathies (DEEs) follows different strategies on one hand including disease‐targeting precision medicine approaches considering the genetic variants and pathomechanisms in DEEs and on the other hand including therapeutic approaches with novel targets or second‐generation drug candidates ...
Heidrun Potschka, Daniel Pérez‐Pérez
wiley   +1 more source

Physical Activity Awareness and Understanding of Treatment Protection Among People With Haemophilia and Their Caregivers in Central Europe

Haemophilia, EarlyView.
ABSTRACT Background Physical activity is now considered generally beneficial for persons with haemophilia (PWH). However, the specific type and extent of activity and its impact on quality of life (QoL) and bleed protection during exercise is under‐researched.
Angelika Batorova, Atanas Banchev, Ana Boban, Brigitte Brand, Melen Brinza, Barbara Faganel Kotnik, Csongor Kiss, Gediminas Puras, Jan Rajnoch, Ester Zapotocka   +9 more
wiley   +1 more source

Despite surge in orphan drug designations, approvals still lag [PDF]

, 2011
Monica Heger
openalex   +1 more source

Orphan Drug Use in Patients With Rare Diseases: A Population-Based Cohort Study. [PDF]

Front Pharmacol, 2022
Gorini F, Santoro M, Pierini A, Mezzasalma L, Baldacci S, Bargagli E, Boncristiano A, Brunetto MR, Cameli P, Cappelli F, Castaman G, Coco B, Donati MA, Guerrini R, Linari S, Murro V, Olivotto I, Parronchi P, Pochiero F, Rossi O, Scappini B, Sodi A, Vannucchi AM, Coi A.   +23 more
europepmc   +1 more source

Clinical/pathological features and survival outcomes of extra‐pulmonary neuroendocrine carcinomas: A retrospective single‐center series

Journal of Neuroendocrinology, EarlyView.
Abstract Poorly differentiated neuroendocrine carcinomas (NECs) are rare malignancies with a dismal prognosis, few therapeutic options, and a lack of predictive factors. We describe a large series of extra‐pulmonary (EP) NEC patients from a neuroendocrine neoplasm (NEN) referral center, aiming to improve the clinical management of these diseases ...
L. Benini, L. Gervaso, S. Frassoni, V. Bagnardi, C. A. Cella, L. Algeri, D. Ciardiello, M. G. Zampino, C. Winchler, S. Boselli, D. Tamayo, F. Spada, N. Fazio   +12 more
wiley   +1 more source

PHP55 RECOMMENDATIONS FOR ORPHAN DRUGS IN TWO EU MEMBER STATES—A FOCUS ON THE UNDERLYING PHARMACOECONOMIC EVALUATIONS

, 2009
Stefan Vegter, R. Postema, KH Tolley, Maarten J. Postma   +3 more
openalex   +1 more source

Assessing the Impacts of the Prescription Drug User Fee Acts (PDUFA) on the FDA Approval Process [PDF]

Congress enacted and renewed the Prescription Drug User Fee Acts (PDUFA) in 1992, and renewed it in 1997 and 2002, mandating FDA performance goals in reviewing and acting on drug applications within specified time periods.
Adrian H. B. Gottschalk, Ernst R. Berndt, Matthew W. Strobeck, Tomas Philipson   +3 more
core  

Narcolepsy and rapid eye movement sleep

Journal of Sleep Research, Volume 34, Issue 2, April 2025.
Summary Since the first description of narcolepsy at the end of the 19th Century, great progress has been made. The disease is nowadays distinguished as narcolepsy type 1 and type 2. In the 1960s, the discovery of rapid eye movement sleep at sleep onset led to improved understanding of core sleep‐related disease symptoms of the disease (excessive ...
Francesco Biscarini, Lucie Barateau, Fabio Pizza, Giuseppe Plazzi, Yves Dauvilliers   +4 more
wiley   +1 more source

Labeling Pediatric Products: BPCA Efforts and Orphan Drug Opportunities. [PDF]

J Pediatr Pharmacol Ther
Wei H, Burckart GJ, Taylor-Zapata P, Wang Y.   +3 more
europepmc   +1 more source

Discrepancies between FDA documents and ClinicalTrials.gov for Orphan Drug-related clinical trial data. [PDF]

PLOS Glob Public Health, 2022
Choudhury MC, Chakraborty I, Saberwal G.
europepmc   +1 more source