Best Practices in SMA
This program will include a discussion of investigational agents not approved by the FDA for use in the United States and data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal.
Overview of SMA
Clinical Characteristics of SMA
Validated Outcome Measures in SMA
Genetic Basis of SMA
SMN2 Copy Number Is a Primary Determinant of Disease Severity
Current and Investigational Targeted Therapies for SMA
Nusinersen
ENDEAR: Study Design
ENDEAR: Endpoints
ENDEAR: Event-Free and Overall Survival
ENDEAR: HINE-2 Responses
ENDEAR: CHOP-INTEND Results
ENDEAR: Safety
Nusinersen in SMA: Early-Phase Studies
CHERISH: Study Design
CHERISH: Results
CHERISH: Results (cont)
NURTURE: Study Design and Results
HINE Motor Milestone Scores Across Studies
Newborn Screening for SMA in the United States
Early Diagnosis and Interventions in SMA
SMN-Targeting Agents in the Pipeline
AVXS-101: Gene Replacement Therapy
AVXS-101: Motor Function Results
AVXS-101: Motor Response in High-Dose Group
Risdiplam: SMN2 Splicing Modifier
Reldesemtiv: Troponin Activator
SRK-015: Myostatin Inhibitor
Moving New Research Into the Clinic
Looking Ahead: Open Research Issues
Abbreviations