Updates in SMA From an International Muscle Meeting

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Presentation transcript:

Updates in SMA From an International Muscle Meeting

This program will include a discussion of data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal.

Spinal Muscular Atrophy Overview

Types of Infant-Onset SMA

Clinical and Economic Burden of SMA

Demographic Profile and Clinical Characteristics Cure SMA Database

Survival Estimates for Type I and Type II SMA

Economic Burden of Type I SMA Retrospective Claims Analysis

Potential Biomarkers for SMA

pNF-H Level as a Biomarker of Disease Activity

pNF-H Levels in SMA

Biomarkers for SMA Clinical Implications

Optimizing Outcomes With Current Strategies for SMA

Survival and Ventilation Use in Type I SMA Analysis of Data From Cure SMA Survey, 2017

Survival and Ventilation Use in Type I SMA Survey Results

Nusinersen

SHINE Study Interim Analysis Patients Transitioned From ENDEAR

SHINE Study Endpoints

SHINE Study Interim Analysis Head Control and Independent Sitting

SHINE Study Interim Analysis Key Learnings

Emerging Clinical Data

AVXS-101

AVXS-101: Phase 1 Study

AVXS-101 Results at 24-Month Follow-Up Age at Dosing and Motor Milestone Achievement

Risdiplam

Risdiplam SMN2 Splicing Modifier

SUNFISH Part 1 Results After 12 Months of Treatment

JEWELFISH Results After 4 Weeks of Treatment

FIREFISH Part 1 Improvement in CHOP-INTEND Scores

FIREFISH Part 1 Achievement of Motor Skills After 8 Months

Branaplam

Branaplam First-in-Human Dose-Escalation Study

Abbreviations