Results 141 to 150 of about 68,732 (285)
European Journal of Haematology, EarlyView.ABSTRACT Objectives The WIL‐31 study demonstrated efficacy and safety of prophylaxis with the plasma‐derived von Willebrand factor/factor VIII concentrate wilate in von Willebrand disease (VWD) of all types and was the only prospective study with an on‐demand run‐in study as an intra‐individual comparator.
Claudia Djambas Khayat +10 more
wiley +1 more source
Epilepsia, EarlyView.Abstract Objective This randomized, double‐blind, phase 1b/2a clinical trial was designed to evaluate the safety, tolerability, and efficacy of oral bexicaserin versus placebo for the treatment of seizures in adolescents and adults with developmental and epileptic encephalopathies (DEEs).
Dennis J. Dlugos +74 more
wiley +1 more source
Epilepsia, EarlyView.Abstract Lennox–Gastaut syndrome (LGS) is one of the most severe, yet one of the most discussed, childhood‐onset developmental and epileptic encephalopathies (DEEs). Dissent among epileptologists on the definition and minimum set of electroclinical features derives from the high etiological heterogeneity within the syndrome, which could make its ...
Antonella Riva +40 more
wiley +1 more source
Epilepsia, EarlyView.Abstract Objective A Pediatric Investigation Plan (PIP) implemented under the European Pediatric Regulation (EC No. 1901/2006) aims to ensure early and systematic evaluation of medicines for children. We analyze PIPs for antiseizure medications (ASMs) submitted to the European Medicines Agency (EMA) since 2006, characterizing their content, focusing on
Valeria Agostini, Stéphane Auvin
wiley +1 more source
Dietary anthocyanidin pelargonidin activates G protein‐coupled receptor 35
The FEBS Journal, EarlyView.Pelargonidin, a red‐fruit‐derived anthocyanidin, was newly identified as a dietary agonist of GPR35, a metabolite‐sensing GPCR implicated in anti‐inflammation. Through screening of dietary compounds, pelargonidin emerged as a potent GPR35 agonist, attenuating inflammation in human intestinal cells.
Fumie Nakashima +11 more
wiley +1 more source
Milestones On Orphan Medicinal Products Development: The 100 First Drugs for Rare Diseases Approved Throughout Europe [PDF]
Clinical Therapeutics, 2015 C. Pontes +8 more
openaire +2 more sources
Hypoxia regulates Th17/Treg balance by altering chromatin accessibility and gene expression
The FEBS Journal, EarlyView.T‐cell differentiation into Th17 or iTreg cells is key to inflammation control. Integrated RNA‐seq and ATAC‐seq analyses reveal that hypoxia induces more extensive gene expression changes in iTreg cells. Elevated levels of Hif1a and Stat3, along with increased accessibility to key transcription factor motifs, may predispose these cells toward ...
Mariana Cázares‐Olivera +5 more
wiley +1 more source
Orphan medical devices: addressing the regulatory and access gaps in the EU and US. [PDF]
Front Public HealthAkodad S, Haon B, Rochaix L, Stevens H.
europepmc +1 more source
Does Misery Love Company? Evidence from pharmaceutical markets before and after the Orphan Drug Act [PDF]
With substantial fixed costs of drug development, more common conditions can support more products. If additional pharmaceutical products are beneficial, they will attract greater consumption and promote better health, e.g. greater longevity.
Frank R. Lichtenberg, Joel Waldfogel
core
HAEMFIX: Impact of Switching From SHL‐FIX to EHL‐FIX in Patients With Haemophilia B
Haemophilia, EarlyView.ABSTRACT Introduction Haemophilia B is an X‐linked recessive bleeding disorder caused by coagulation factor IX (FIX) deficiency. Treatment involves intravenous replacement of FIX. Recently, extended half‐life (EHL) FIX products have been introduced alongside standard half‐life (SHL) products to optimize therapy. Aim This study evaluated bleeding rates,
Jasmin Lonardi +11 more
wiley +1 more source