Results 191 to 200 of about 1,419,402 (298)

Real‐World Outcomes of Prophylaxis With rIX‐FP in Germany: A Prospective, Non‐Interventional Study in Haemophilia B

Haemophilia, EarlyView.
ABSTRACT Introduction Efficacy and safety of albutrepenonacog alfa (rIX‐FP) in people with haemophilia B (PwHB) has been previously demonstrated in clinical trials. However, real‐world data insights are required. Aim To describe real‐world effectiveness and tolerability of rIX‐FP in PwHB in Germany.
Johannes Oldenburg, Swee Wenning, Susanne Holzhauer, Wolfgang Miesbach, Robert Klamroth, Thomas Linhoff, Patrick Sommerer, Christian Pfrepper, Martin Olivieri, all study investigators   +9 more
wiley   +1 more source

Uttroside B, a US FDA-designated 'Orphan Drug', mitigates the development of hepatocellular carcinoma and its pulmonary metastasis via EGFR/ERK-mediated inhibition of SREBP-1 and STAT-3. [PDF]

Cell Death Discov
Keerthana CK, Rayginia TP, Kalimuthu K, Saha S, Haritha NH, Nisthul Areekkara A, Swetha M, Aiswarya SU, Nath LR, Varma SS, Viswanathan A, S J, J S, Js A, Praveen A, Jaikumar VS, Sundaram S, Anto NP, Maiti TK, Sadasivan C, Isakov N, Lankalapalli RS, Harikumar KB, Anto RJ.   +23 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

Next Generation Smart Implant Surfaces: Engineering Multifunctional Antimicrobial, Immunomodulatory and Regenerative Interfaces

Journal of Periodontal Research, EarlyView.
Natural‐inspired nano‐textured implant surfaces can be functionalised to modulate macrophage polarisation and local immune responses. This bioactive control promotes improved tissue integration and healing outcomes. ABSTRACT Dental implants rely on a stable and functional interface between the implant surface, surrounding tissues, and the oral ...
Roberto S. Pessoa, Lauren E. Steele, Mehvish Mumtaz, Denver P. Linklater, Neil M. O'Brien‐Simpson, Vladimir Baulin, Peter Kingshott, Roy Judge, Alastair J. Sloan, Elena P. Ivanova   +9 more
wiley   +1 more source

Orphan drug policy analysis in China. [PDF]

Front Pharmacol
Liu M, Lu Y, Li J, Zhang Y, Zhang S, Liu Y.   +5 more
europepmc   +1 more source

A systematic review of moral reasons on orphan drug reimbursement. [PDF]

Orphanet J Rare Dis, 2021
Zimmermann BM, Eichinger J, Baumgartner MR.   +2 more
europepmc   +1 more source

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

Diagnostic Value of Psychomotor Vigilance Task for Severe Sleep Inertia in Idiopathic Hypersomnia Versus Other Sleep Disorders Without Sleep Inertia

Journal of Sleep Research, EarlyView.
Diagnostic Value of PVT for Severe Sleep Inertia in Idiopathic Hypersomnia. ABSTRACT Sleep inertia impairs performance upon awakening and may be assessed using the psychomotor vigilance task (PVT). This study aimed to determine optimal PVT lapse cut‐offs at awakening to objectively measure sleep inertia by comparing patients with idiopathic hypersomnia
Elisa Evangelista, Alexandre Derré, Lucie Barateau, Anna Laura Rassu, Sofiene Chenini, Isabelle Jaussent, Yves Dauvilliers   +6 more
wiley   +1 more source

Global insight into rare disease and orphan drug definitions: a systematic literature review. [PDF]

BMJ Open
Abozaid GM, Kerr K, Alomary H, Al-Omar HA, McKnight A.   +4 more
europepmc   +1 more source

Postmortem Evidence of CRH Neuron Reduction in Narcolepsy Without Cataplexy With Borderline Hypocretin‐1 Levels

Journal of Sleep Research, EarlyView.
ABSTRACT Narcolepsy is classified as type 1 (NT1) or type 2 (NT2) mainly according to hypocretin deficiency rather than cataplexy. While CRH neuron loss in the PVN has been described in NT1, it remains unclear whether similar changes occur in narcolepsy without cataplexy.
Ling Shan, Suzan Linssen, Rolf Fronczek, Lucie Barateau, Christelle Peyron, Yves Dauvilliers   +5 more
wiley   +1 more source