An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Muscular dystrophies are a wide range of muscle disorders related to the congenital or genetically determined absence of certain essential muscle fiber components (e.g., dystrophin). In most patients, ...

The study is published in Stem Cell Research & Therapy. Ullrich congenital muscular dystrophy (UCMD) is an early-onset, progressive muscular disease characterized by muscle weakness and joint ...

An Indiana man is proving that determination can break down barriers by starting his own company despite life's challenges.

Posters presented at the 2025 Muscular Dystrophy Association ... excluding those with congenital myotonic dystrophy. Participants were required to have continuous data activity for at least ...

Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach ...

We report on a preterm neonate of 30 weeks gestational age who presented with marked muscular hypotonia ... at birth and was diagnosed with congenital myotonic dystrophy. Neuroimaging at 36 ...

At this week’s Muscular Dystrophy Association Clinical and Scientific Conference in Dallas, researchers from Suzhou Genassist Therapeutics Co. Ltd. presented preclinical data for GEN-6050X ...