Results 161 to 170 of about 169,015 (342)

Pain Experiences and Prescription Pain Medications Among People With Selected Muscular Dystrophies in the Muscular Dystrophy Surveillance, Tracking, and Research Network

Muscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Pain is a recognized symptom of muscular dystrophy (MD), but little is known about prescription pain medications in this population. We describe pain experiences and pain medications prescribed for individuals with selected MDs using population‐based surveillance data collected by the Muscular Dystrophy Surveillance, Tracking,
Jonathan Suhl, Kristin M. Conway, Shiny Thomas, Sonja A. Rasmussen, James F. Howard, Nicholas E. Johnson, Paul A. Romitti, Katherine D. Mathews, The Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet)   +9 more
wiley   +1 more source

A model‐informed clinical trial simulation tool with a graphical user interface for Duchenne muscular dystrophy

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, Juan Francisco Morales, Sanghoon Kang, Marian Klose, Rebecca J. Willcocks, Michael J. Daniels, Ramona Belfiore‐Oshan, Glenn A. Walter, William D. Rooney, Krista Vandenborne, Sarah Kim   +10 more
wiley   +1 more source

Single cell RNA sequencing of human FAPs reveals different functional stages in Duchenne muscular dystrophy

Frontiers in Cell and Developmental Biology
Background: Duchenne muscular dystrophy is a genetic disease produced by mutations in the dystrophin gene characterized by early onset muscle weakness leading to severe and irreversible disability. Muscle degeneration involves a complex interplay between
Esther Fernández-Simón, Patricia Piñol-Jurado, Rasya Gokul-Nath, Adrienne Unsworth, Jorge Alonso-Pérez, Marianela Schiava, Andres Nascimento, Andres Nascimento, Giorgio Tasca, Rachel Queen, Dan Cox, Xavier Suarez-Calvet, Xavier Suarez-Calvet, Jordi Díaz-Manera, Jordi Díaz-Manera, Jordi Díaz-Manera   +15 more
doaj   +1 more source

Serum enzyme studies in muscle disease: Part II Serum creatine kinase activity in muscular dystrophy and in other myopathic and neuropathic disorders [PDF]

, 1964
John Pearce, R. J. Pennington, J. N. Walton   +2 more
openalex   +1 more source

A computational tool to optimize clinical trial parameter selection in Duchenne muscular dystrophy: A practical guide and case studies

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, Varun Aggarwal, Mike Pauley, Diane Corey, Daniela J. Conrado, Karthik Lingineni, Juan Francisco Morales, Deok Yong Yoon, Yi Zhang, Zihan Cui, Jackson Burton, Jane Larkindale, Shu Chin Ma, Collin Hovinga, Terina Martinez, Klaus Romero, Ramona Belfiore‐Oshan, Sarah Kim, the Duchenne Regulatory Science Consortium (D‐RSC) and the CINRG DNHS investigators   +18 more
wiley   +1 more source

Intellectual Impairment in Muscular Dystrophy [PDF]

, 1965
Victor Dubowitz
openalex   +1 more source

Subcutaneous Foslevodopa as Novel Rescue Therapy for Akinetic Crisis in Parkinson's Disease: A Case Report

Movement Disorders Clinical Practice, EarlyView.
Bendix Labeit, Julius Schulten, Alfons Schnitzler, Christian Johannes Hartmann   +3 more
wiley   +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, Mamata Karmacharya, Hyunmin Choi, Sumit Kumar, Yoon‐Kyoung Cho   +4 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

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, Nei Li, Yudian Xiao, Rohan Palanki, Hannah Yamagata, Michael J. Mitchell, Xuexiang Han   +6 more
wiley   +1 more source