Treatments Approved for Treatment of Spinal Muscular Atrophy
Key opinion leaders (KOLs) examine the mechanisms of action and other pertinent information regarding FDA-approved treatments for spinal muscular atrophy (SMA), sharing their perspectives on these therapies with a particular focus on the various modes of administration.
EP: 1.Clinical Features and Phenotypes of Spinal Muscular Atrophy
EP: 2.Natural History of Spinal Muscular Atrophy
EP: 3.Presentation of Adult Spinal Muscular Atrophy in Clinics
EP: 4.Transitions of Care: Moving Spinal Muscular Atrophy Patients from Pediatric/Adolescent Care to Adult Clinics
EP: 5.Key Players in Transitioning Patients with SMA from Pediatric Care to Adult Health Care
EP: 6.Managing Clinical Stability in Adults Spinal Muscular Atrophy Patients
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EP: 7.Treatments Approved for Treatment of Spinal Muscular Atrophy
EP: 8.Factors Guiding Treatment Selection in Adult Spinal Muscular Atrophy
- Please review mechanism of action and other details for treatments that are FDA approved for SMA.
- Nusinersen, Onasemnogene abeparvovec, Risdiplam
- Based on the pathophysiology of SMA, do you expect mechanism of action to impact disease pathology differently?
How do you feel about mode of administration?
How do you discuss the progressive nature of SMA with your adult patients?
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