Results 161 to 170 of about 54,763 (333)
CPT: Pharmacometrics &Systems Pharmacology, EarlyView.Abstract Quantitative model‐based clinical trial simulation tools play a critical role in informing study designs through simulation before actual execution. These tools help drug developers explore various trial scenarios in silico to select a clinical trial design to detect therapeutic effects more efficiently, therefore reducing time, expense, and ...
Jongjin Kim +10 more
wiley +1 more source
CPT: Pharmacometrics &Systems Pharmacology, EarlyView.Abstract Duchenne muscular dystrophy (DMD), a rare pediatric disease, presents numerous challenges when designing clinical trials, mainly due to the scarcity of available trial participants and the heterogeneity of disease progression. A quantitative clinical trial simulator (CTS) has been developed based on previously published five disease ...
Jordan Wilk +18 more
wiley +1 more source
Clinical and pathological study of a case of congenital muscular dystrophy [PDF]
, 1966 S. S. Gubbay, J. N. Walton, G. W. Pearce
openalex +1 more source
Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing
Small, EarlyView.The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa +4 more
wiley +1 more source
DNA diagnostic tests in Xp21 dystrophy families for prenatal diagnosis
The Turkish Journal of Pediatrics, 1998 Duchenne and Becker muscular dystrophies are X-linked genetic disorders characterized by dystrophin gene defects. We have studied 250 families with Duchenne and Becker muscular dystrophies (D/BMD) by molecular genetic methods since 1992.
P Dinçer, H Topaloğlu, S Ayter
doaj
Limb-Girdle Muscular Dystrophies Classification and Therapies. [PDF]
J Clin Med, 2023 Bouchard C, Tremblay JP.
europepmc +1 more source
Alpha-hydroxybutyrate dehydrogenase activity in sex-linked muscular dystrophy [PDF]
, 1966 H. A. Johnston +3 more
openalex +1 more source
Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components
Small Methods, EarlyView.The review presents a comprehensive overview of each component of lipid nanoparticles（LNPs）and their effects on editing efficiency. It specifically highlights strategies for achieving non‐liver delivery, aiming for broader applications in gene editing. Furthermore, this review summarizes the applications of LNPs in gene editing and offers insights for ...
Fan Wu +6 more
wiley +1 more source