The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children from age 4 through 5 years of age. Pediatric patients who ...

New research from the Exercise Medicine Research Institute at Edith Cowan University (ECU) has highlighted that children undergoing cancer treatment often lose skeletal muscle at a time when they ...

The greatest reduction in skeletal muscle mass was in the lower extremities, a new report shows. Low skeletal muscle mass may be an early manifestation of fabry disease (FD), according to a new report ...

Although a profound transformation has occurred in both survival and clinical course, long-term data on the efficacy and safety of disease-modifying therapies is required to inform their use in ...

A lightweight robotic device that facilitates neuromuscular recovery in children with spinal muscular atrophy (SMA), helping them to stand unassisted, is published in Nature this week. Improved ...

Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...

An Australian-first biobank will be established to improve and discover new treatments for children with genetic muscle diseases. These diseases, spanning dystrophies and myopathies, are characterised ...

New research published in JAMA recommends daily steroid doses for children with Duchenne muscular dystrophy (DMD), marking a significant change in how the disease is treated. University of Rochester ...

Gene therapies have extended survival in muscular dystrophy, requiring improved care transitions from pediatric to adult systems. Current systems lack adequate staffing, specialists, and communication ...

On March 27, 2024, Ann & Robert H. Lurie Children's Hospital of Chicago treated its first patient with ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy for Duchenne muscular ...

Muscular dystrophy is a group of diseases that make muscles weaker and less flexible over time. It is caused by a problem in the genes that control how the body keeps muscles healthy. Duchenne ...

Researchers evaluated oxygen saturation and arterial CO2 variables to screen for sleep disordered breathing in children with SMA type 1-3. Of the measures evaluated, only an oxygen desaturation index ...