The Importance of Early Intervention in Duchenne Muscular Dystrophy
June 18th 2025A panelist discusses how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies like exon skipping or gene therapy, and consistent monitoring by a multidisciplinary clinical team.
The Role of Gene Therapy for Patients with Duchenne Muscular Dystrophy
June 18th 2025A panelist discusses how gene therapy using microdystrophin delivered through AAV vectors shows promise for DMD treatment, though challenges remain with delivery efficiency and determining which patients will benefit most.
Understanding the Causes of Duchenne Muscular Dystrophy
June 4th 2025A panelist discusses how steroid use in DMD patients provides significant benefits in delaying disease progression, particularly loss of ambulation, despite potential side effects including mood disorders, bone weakness, and weight gain.