Results 101 to 110 of about 4,658,239 (268)
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective To clarify the clinical relevance of dopamine transporter single‐photon emission computed tomography (DAT‐SPECT) abnormalities in amyotrophic lateral sclerosis (ALS), with a prespecified focus on sex‐stratified associations with disease progression and short‐term prognosis.
Tomoya Kawazoe +7 more
wiley +1 more source
Journal of Blood MedicineZoran Igrutinović,1,2 Hélène Louise Hooimeijer,3 Karim Kentouche,4 Jaco Botha,5 Peter L Turecek,6 Marta Kokot-Kierepa,5 Hanna T Gazda7 1Clinic of Pediatrics, Department of Hemato-Oncology, University Clinical Center of Kragujevac, Kragujevac ...
Igrutinović Z +6 more
doaj
New developments in the management of moderate-to-severe hemophilia B
Journal of Blood Medicine, 2016 Moniba Nazeef,1,2 John P Sheehan1,2 1Department of Medicine, Division of Hematology/Oncology, 2UW Carbone Cancer Center, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA Abstract: Hemophilia B is an X-linked genetic ...
Nazeef M, Sheehan JP
doaj
The molecular mechanism of factor IX activation by factor XIa
, 2009 Activation of coagulation factor IX is a pivotal reaction in the formation and maintenance of a fibrin clot. Factor IX is activated by two structurally unrelated enzymes; factor XIa of the intrinsic coagulation cascade and the extrinsic cascade complex ...
Smith, Stephen Bradford
core
The Arg-4 mutant factor IX Strasbourg 2 shows a delayed activation by factor XIa
, 1993 We have characterized at the DNA and protein levels a mutant factor IX, factor IX Strasbourg 2, which is responsible for a severe form (< 0.01 U/ml) of haemophilia B. Factor IX Strasbourg 2 has a higher molecular weight than normal factor IX.
Ohlmann, Philippe +10 more
core +3 more sources
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Facioscapulohumeral muscular dystrophy (FSHD) is one of the most debilitating and common muscular dystrophies. Despite its severity, no approved therapy exists for FSHD patients. However, several therapeutic candidates are currently under development, and some have recently entered clinical trials, marking the need for reliable ...
Mustafa Bilal Bayazit +11 more
wiley +1 more source
Extended Half-Life Factor VIII and Factor IX Preparations
, 2018 In the last couple of years, several extended half-life factor VIII and factor IX preparations were intensively studied and gained approval. In order to extend half-lives, techniques like fusion to protein conjugates (Fc part of IgG<sub>1</sub ...
Lukas Graf
core +1 more source
Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito +8 more
wiley +1 more source
Coagulopathy, Factor IX Deficiency
, 2010 McLoughlin, T.(2010). Coagulopathy, Factor IX Deficiency. Essence of Anesthesia Practice.(pp.88).
McLoughlin, Thomas, MD
core +1 more source
Uncovering G Protein‐Coupled Receptors: Novel Targets and Biomarkers for Predicting Glioma Prognosis
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Background Low‐grade gliomas (LGG) exhibit significant heterogeneity and recurrence risk. G protein‐coupled receptors (GPCR) contribute to glioma malignant progression, but their prognostic value remains unclear. This work attempts to formulate a GPCR‐based outcome‐predicting model for LGG. Methods Based on TCGA LGG data, the enrichment scores
Jun Yang +4 more
wiley +1 more source