New Horizons for SMA

This program will include a discussion of investigational agents not approved by the FDA for use in the US, and data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal.

Introduction to Spinal Muscular Atrophy

Introduction to Spinal Muscular Atrophy (cont)

Why Treat SMA?

Burden of SMA Type 1

Burden of SMA Types 2 and 3

Clinical Trial Endpoints

Assessing Outcomes in Clinical Practice

Mechanisms of Action of New Therapies

SMA Treatment Mechanism of Action

SMA Treatment Mechanism of Action (cont)

Nusinersen

ENDEAR Trial Design

ENDEAR Trial Results

ENDEAR: Motor Milestone Responders

ENDEAR: CHOP INTEND Motor Function

Change in HINE Score From Baseline

Change in CHOP-INTEND Score From Baseline

CHERISH Trial Design

CHERISH Trial: HFMSE Scores

Phase 1 Trial of AVXS-101

Phase 1 Trial of AVXS-101 (cont)

Efficacy in Phase 1 Trial of AVXS-101

Olesoxime: Mechanism of Action

Olesoxime: Efficacy and Safety

Maintaining Motor Function in SMA

Agents That May Improve Nerve or Muscle Function

Conclusions

Abbreviations