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Standards of Care in Duchenne Muscular Dystrophy: A 2018 Update

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Presentation on theme: "Standards of Care in Duchenne Muscular Dystrophy: A 2018 Update"— Presentation transcript:

1 Standards of Care in Duchenne Muscular Dystrophy: A 2018 Update

2 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.

3 Defining DMD

4 Prevalence and Burden of DMD

5 DMD Inheritance

6 DMD Mutations

7 DMD Reading Frame Disruption

8 Early Signs and Symptoms of DMD

9 Progression of DMD

10 Tests to Confirm the Diagnosis of DMD

11 Coordinated Care Model for DMD

12 Proactive Rather Than Reactive Respiratory Care for DMD

13 2016 AAN Guidelines for Corticosteroid Treatment of DMD

14 2016 AAN Guidelines for Corticosteroid Treatment of DMD (cont)

15 2016 AAN Guidelines for Corticosteroid Treatment of DMD (cont)

16 Corticosteroids Underused in Clinical Practice

17 Disease-Modifying Antisense Oligonucleotide

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

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

20 Conclusions

21 Abbreviations


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