Standards of Care in Duchenne Muscular Dystrophy: A 2018 Update

This program will include a discussion of off-label treatment and 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.

Defining DMD

Prevalence and Burden of DMD

DMD Inheritance

DMD Mutations

DMD Reading Frame Disruption

Early Signs and Symptoms of DMD

Progression of DMD

Tests to Confirm the Diagnosis of DMD

Coordinated Care Model for DMD

Proactive Rather Than Reactive Respiratory Care for DMD

2016 AAN Guidelines for Corticosteroid Treatment of DMD

2016 AAN Guidelines for Corticosteroid Treatment of DMD (cont)

2016 AAN Guidelines for Corticosteroid Treatment of DMD (cont)

Corticosteroids Underused in Clinical Practice

Disease-Modifying Antisense Oligonucleotide

Three-Part Evidence-Based Guidelines for DMD Diagnosis and Treatment

Three-Part Evidence-Based Guidelines for DMD Diagnosis and Treatment (cont)

Conclusions

Abbreviations