Managing Spinal Muscular Atrophy in Adult Patients : Episode 13

Opinion

Video

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

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.

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

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

Discuss real-world evidence for risdiplam in adult patients.

Related Videos

Marcello Moccia, MD, PhD

Mikael Cohen, MD

Wallace Brownlee, MBChB, PhD, FRACP

Tom Fuchs, MD, PhD

Valentin Krüger, MD

Shamik Bhattacharyya, MD

Phillip Kuo, MD

Igor Koralnik, MD

Related Content

Related Content

Do Not Sell My Information

Terms and Conditions

Contact Info

2 Commerce Drive
Cranbury, NJ 08512

© 2025 MJH Life Sciences

All rights reserved.