Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

Introduction Duchenne muscular dystrophy (DMD) is one of the most severe and devastating
neuromuscular hereditary diseases with a male newborn incidence of 20 000 cases each …

Duchenne muscular dystrophy (DMD) is a devastating disease that results in life-limiting
complications such as loss of skeletal muscle function as well as respiratory and cardiac …

The prediction of transgene product expression in human is important to guide first-in-
human (FIH) dose selection for viral vector-based gene replacement therapies. Recently …

Aim To systematically evaluate the diagnostic accuracy of the creatine kinase isoenzyme-
MM (CK-MM) test in newborn screening for Duchenne Muscular Dystrophy (DMD). Methods …

Background To systematically evaluate the diagnostic accuracy of the creatine kinase
isoenzyme-MM (CK-MM) test in newborn screening for Duchenne muscular dystrophy …

Abstract Duchenne Muscular Dystrophy (DMD) is a genetic disorder involving progressive
muscle deterioration leading to loss of mobility, cardiomyopathy, and respiratory …

The identification of the genes causing the most common muscular dystrophies over the past
few decades has allowed the design of new classes of targeted therapeutics. A number of …

The carbohydrate antigen Sda is expressed on cells and secretions of the vast majority of
Caucasians. The epitope is formed by a terminal GalNAc residue β4-linked to a α3 …