Duchenne and Becker Muscular Dystrophy

The coronavirus disease 2019 (COVID‐19) pandemic has resulted in the reorganization of health‐care settings affecting clinical… Expand

PurposePatient preference information (PPI) have an increasing role in regulatory decision-making, especially in benefit–risk… Expand

Background The social context of rare disease research is changing, with increased community engagement around drug development… Expand

Manifestations of Duchenne and Becker muscular dystrophy (DBMD) are present in up to 40% of heterozygous females, but there are… Expand

Introduction: In progressive conditions, such as Duchenne and Becker muscular dystrophy (DBMD), the need for care may outpace… Expand

Duchenne and Becker muscular dystrophy (DBMD) are allelic, X‐linked recessive, neuromuscular disorders characterized by… Expand

Duchenne and Becker muscular dystrophy (DMD/BMD) are X-linked recessive disorders caused by mutation in dystrophin gene. We… Expand

JPGN Volume 53, N N onalcoholic fatty liver disease (NAFLD) encompasses a wide spectrum of conditions ranging from simple… Expand

Objective. The goal was to assess women's knowledge and heart health behaviors consistent with the American Academy of Pediatrics… Expand