Reports world wide Duchenne Muscular Dystrophy (DMD) | Page 2

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Major Table of Contents
1 KEY TAKEAWAYS
2 DISEASE BACKGROUND
3
4
5
6
TREATMENT
Corticosteroids
Genetic therapies
Vitamin D supplement
7 EPIDEMIOLOGY
8 MARKETED DRUGS
9 Approvals by country
10 PIPELINE DRUGS
11 KEY UPCOMING EVENTS
12
13
14
15
16
17
18 KEY REGULATORY EVENTS
Exondys Revisited? Translarna Brings Efficacy Woes Into US Panel Review
Before Price Controversy, Emflaza Got Easy US Nod With Old But Good Enough Data
Sarepta's Exondys 51 (Eteplirsen) Approved After Contentious Internal FDA Debate
Marathon Looking To Go The Distance In DMD
Scots Hammer PTC's Translarna As EU Reviews Conditional Approval
FDA Refuses To File Translarna NDA; PTC Plummets
19
20
21
22
23
24 LICENSING AND ASSET ACQUISITION DEALS
Lucky No. 35? Sarepta And BioMarin Settle Global Patent Disputes
Bristol Licenses Phase II DMD Candidate To Roche
Sarepta Snaps Up Gene Therapy Approaches to DMD
Sarepta’s $852m Summit Collaboration Could Enable DMD Drug Combinations
PARENT PATENTS
25 REVENUE OPPORTUNITY
26
27
28
29
CLINICAL TRIAL LANDSCAPE
Sponsors by status
Sponsors by phase
Recent events
30 BIBLIOGRAPHY
31 Prescription information
32 APPENDIX
LIST OF FIGURES