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
How we count matters - infections are a major cause of death among patients with type 2 diabetes. [PDF]
Støvring H, Wild S.
europepmc +1 more source
ABSTRACT Objective Recurrent high‐grade gliomas have a poor prognosis and limited therapeutic options. This study aimed to evaluate the safety and efficacy of SYHA1813, a dual inhibitor of VEGFR and CSF1R, in patients with recurrent high‐grade gliomas.
Zhuang Kang+16 more
wiley +1 more source
The contribution of minimally invasive tissue sampling compared to antemortem-derived cause of death determination among inpatient child deaths: the minimally invasive tissue sampling in Malawi study. [PDF]
Voskuijl WP+18 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
Improving medical certification of cause of death in Assiut University Children Hospital: an intervention study. [PDF]
El-Gibaly O+3 more
europepmc +1 more source
Metabolic Consequences of Rheumatoid Arthritis
Patients with rheumatoid arthritis (RA) may have metabolic disruption, which can contribute to adverse long‐term outcomes, for multiple reasons. Patients with RA appear to have a higher risk of sarcopenia, type 1 and type 2 diabetes mellitus, metabolic syndrome, and hypertension. Systemic inflammation in RA can cause a “lipid paradox,” with reduced low‐
Stevie Barry+2 more
wiley +1 more source
Improving completeness & reducing errors in medical certification of cause of death: The impact of electronic mortality software in a tertiary care centre in South India. [PDF]
Thanigasalam A, Ramu R, Rajarethinam K.
europepmc +1 more source
Objective We estimated the real‐world cost‐effectiveness of a standardized education and exercise therapy program (GLA:D) compared to usual care (UC) for people managing hip and/or knee osteoarthritis (HKOA). Methods We used a prospective matched cohort design to recruit people (aged >45 years) diagnosed with HKOA who used GLA:D or UC (not on a ...
Darren R. Mazzei+4 more
wiley +1 more source