Natural History of Spinal Muscular Atrophy

Author(s):

Lauren Elman, MD,Arun S. Varadhachary, MD, PhD

Panelists examine the natural history of spinal muscular atrophy (SMA), assessing the characteristic indicators of motor function deterioration and the loss of ambulatory abilities as patients advance in age.

EP: 1.Clinical Features and Phenotypes of Spinal Muscular Atrophy

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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

EP: 7.Treatments Approved for Treatment of Spinal Muscular Atrophy

EP: 8.Factors Guiding Treatment Selection in Adult Spinal Muscular Atrophy

EP: 9.Switching Treatments in Spinal Muscular Atrophy

EP: 10.Determining Treatment Success in Spinal Muscular Atrophy

EP: 11.Counseling Patients About Treatment Outcomes in Spinal Muscular Atrophy

EP: 12.Real-World Data and Stability with Nusinersen in Adult Patients with Spinal Muscular Atrophy

EP: 13.Real-World Data for Risdiplam in Adult Patients with Spinal Muscular Atrophy

EP: 14.Transition of Care in Young Adults With Spinal Muscular Atrophy

EP: 15.Recommendations for Clinicians Taking Care of Patients with SMA

What do we know about the natural history of SMA?
Discuss decline in motor function and loss of ambulation over time in adults.