Researchers are investigating the safety and effectiveness of a new gene therapy product that replaces the defective β-sarcoglycan gene.
Researchers are investigating the safety and effectiveness of a new gene therapy product that replaces the defective β-sarcoglycan gene.
While endocrinologists are not always part of a core DMD treatment team, their expertise is necessary, especially in handling the effects of long-term glucocorticoid therapy.
A meta-analysis shows people who have experienced concussion and/or mild traumatic brain injury are more likely to attempt suicide and to have suicidal ideation than people who have not experienced the injuries.
Surgical management can achieve seizure freedom in pediatric patients with drug-resistant epilepsy, but special precautions must be taken.
Genome-wide association study identifies two variants that may yield therapies for children with DMD.
Study also indicates early treatment may provide best outcomes.