重點精華時間軸

00:00 Introduction to Spinal Muscular Atrophy International Case Experiences and Updates
05:40 Case 1 (video): Patient with tracheostomy, improve to BiPAP at night after gene therapy at 9 months old
11:30 Case 2 (video): Malnourished patient status post nusinersen, weight gain after switching to gene therapy at 18 months old
15:25 Case 3 (video): Steroid adjustment for transaminitis following gene therapy
21:05 Case 4 (video): Asymptomatic patient with 2 copies of SMN2 gene received gene therapy and normally grow up to 17 months old
24:20 SMA newborn screening economic analysis worldwide
27:00 Gene therapy cohort studies: motor, ventilation, and bulbar status
31:35 Multidisciplinary SMA care team follow-up and parental education
33:20 Q&A: What's the expected prognosis for asymptomatic type 1 SMA treated before 2 years old?
34:30 Q&A: How to evaluate the need for gastrostomy?
36:50 Neonatal Symptomatic SMA Treated with Onasemnogene abeparvovec: One-year-old Outcome Evaluation in Taiwan (video)