Panel Discussion: Patient Perspectives on Involvement in Gene Therapy Trials for Muscular Disorders

Time: 1:30 pm
day: Day One


  • The potential for platform studies – Allowing patients to pursue further therapies without compromising the long-term results of the clinical studies
  • Improving the clarity regarding label restrictions and age at dosing
  • Discussing the importance of caregiver advocacy for gene therapy patients with muscular disorders