Long‐Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons
ABSTRACT Objectives This ongoing, open‐label extension study is evaluating the long‐term safety, tolerability, and efficacy of givinostat, a Class I and II histone deacetylase inhibitor, in patients with Duchenne muscular dystrophy (DMD). Methods The recruited patients completed one of two prior clinical studies (one Phase 2 and one Phase 3 [EPIDYS ...
Craig M. McDonald +74 more
wiley +1 more source
High-Accuracy Quantitative Nuclear Magnetic Resonance Using Improved Solvent Suppression Schemes. [PDF]
Garrido BC +3 more
europepmc +1 more source
Durability of Response to B‐Cell Maturation Antigen‐Directed mRNA Cell Therapy in Myasthenia Gravis
ABSTRACT Objective We report the 12‐month follow‐up outcomes from a Phase 2 clinical trial (NCT04146051) evaluating Descartes‐08, a BCMA‐directed RNA chimeric antigen receptor T‐cell (rCAR‐T) therapy for refractory generalized myasthenia gravis (MG).
Nizar Chahin +10 more
wiley +1 more source
Energy-Degenerate Photon-Pair Generation from Individual CsPbBr<sub>3</sub> Quantum Dots. [PDF]
Zhu C +6 more
europepmc +1 more source
THE ACTIVE AREA IN COURSE OF EXCITATION CONDUCTION (OBSERVATIONS ON LILLIE'S NERVE MODEL)
Kazumi Yamagiwa
openalex +2 more sources
NR4A1 Exerts Pro‐Tumor Role in Glioblastoma via Inducing xCT/GPX4‐Regulated Ferroptosis
ABSTRACT Purpose This study investigates NR4A1's paradoxical roles in glioblastoma (GBM) progression, focusing on its mechanistic link to ferroptosis regulation. We aimed to resolve conflicting reports of NR4A1 as both an oncogene and a tumor suppressor by defining its transcriptional control over xCT/GPX4‐mediated iron homeostasis and its clinical ...
Peng Tao +10 more
wiley +1 more source
Giant two-photon upconversion from 2D exciton in doubly-resonant plasmonic nanocavity. [PDF]
Liu F +13 more
europepmc +1 more source
Chronic Pain in Parkinson’s Disease: Prevalence, Sex Differences, Regional Anatomy and Comorbidities
Chronic pain affected 66.2% of 10,631 individuals with Parkinson's disease, with higher prevalence and severity in females. Pain most often involved the buttocks, lower back, neck, and knees, and was linked to depression, sleep disorders, and osteoarthritis.
Natalia S. Ogonowski +13 more
wiley +1 more source
Velocity Map Imaging of NO (A) from Photodissociation of the Ar-NO Complex. [PDF]
Parsons BF +3 more
europepmc +1 more source

