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Opinion|Videos|July 22, 2024

Real-World Data for Risdiplam in Adult Patients with Spinal Muscular Atrophy

Author(s)Lauren Elman, MD, Arun S. Varadhachary, MD, PhD , Thomas Crawford, MD

Panelists examine the available real-world data (RWD) on the use of risdiplam in adult patients with spinal muscular atrophy (SMA) from various studies conducted worldwide, discussing its potential efficacy and safety as a treatment option for adult SMA patients.

Episodes in this series

Video 1 - 5 KOLs are featured in "Clinical Features and Phenotypes of Spinal Muscular Atrophy"

EP. 1 Clinical Features and Phenotypes of Spinal Muscular Atrophy

Video 2 - 5 KOLs are featured in "Natural History of Spinal Muscular Atrophy"

EP. 2 Natural History of Spinal Muscular Atrophy

Video 3 - 5 KOLs are featured in "Presentation of Adult Spinal Muscular Atrophy in Clinics"

EP. 3 Presentation of Adult Spinal Muscular Atrophy in Clinics

Video 3 - 5 KOLs are featured in "Transitions of Care: Moving Spinal Muscular Atrophy Patients from Pediatric/Adolescent Care to Adult Clinics"

EP. 4 Transitions of Care: Moving Spinal Muscular Atrophy Patients from Pediatric/Adolescent Care to Adult Clinics

Video 5 - 5 KOLs are featured in "Key Players in Transitioning Patients with SMA from Pediatric Care to Adult Health Care"

EP. 5 Key Players in Transitioning Patients with SMA from Pediatric Care to Adult Health Care

Video 5 - 5 KOLs are featured in "Managing Clinical Stability in Adults Spinal Muscular Atrophy Patients"

EP. 6 Managing Clinical Stability in Adults Spinal Muscular Atrophy Patients

5 KOLs are featured in this series.

EP. 7 Treatments Approved for Treatment of Spinal Muscular Atrophy

5 KOLs are featured in this series.

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

5 KOLs are featured in this series.

EP. 9 Switching Treatments in Spinal Muscular Atrophy

5 KOLs are featured in this series.

EP. 10 Determining Treatment Success in Spinal Muscular Atrophy

5 KOLs are featured in this series.

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

5 KOLs are featured in this series.

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

5 KOLs are featured in this series.

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

5 KOLs are featured in this series.

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

5 KOLs are featured in this series.

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

Discuss real-world evidence for risdiplam in adult patients.

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