Panel Discussion: Patient Perspectives on Involvement in Gene Therapy Trials for Muscular Disorders
Time: 1:30 pm
day: Day One
Details:
- 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
