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Review Of Phase II And Phase III Orphan Drug Clinical Trials For Duchenne Muscular Dystrophy: Journal Article

February 10, 2013

Expert Opinion on Orphan Drugs is a new international, peer-reviewed journal that covers the topic of R&D for rare diseases and orphan drugs. The Editor-In-Chief is Prof. M. Ian Phillips, Director of the Center for Rare Disease Therapies at the Keck Graduate Institute of Applied Life Sciences, in California. The inaugural issue, the January 2013 (Vol. 1, No. 1) issue, is available online.

A FREE article from this issue, discusses and reviews Phase II and Phase III clinical trials for Duchenne Muscular Dystrophy (DMD). The authors review evidence-based studies for the current status of treatments for DMD. The following therapeutic interventions are evaluated and compared :

1) Corticosteroids

2) Myostatin Inhibitors (i.e. Milo BioTechnology’s orphan drug for Follistatin Therapy)

3) Phosphodiesterase Inhibitors

4) Insulin Growth Factor 1 (IGF-1)

5) Nutritional Intervention

6) Stop Codon Read-Through (i.e. PTC Therapeutics’ orphan drug Ataluren)

7) Exon Skipping (i.e. Sarepta Therapeutics’ orphan drug Eteplirsen & Prosensa’s orphan drug Drisapersen)

8) Gene Therapy.

Table I (“Summary of current therapeutic interventions evaluated for DMD”) on pages 39 – 40, summarizes the current landscape for Phase II and III clinical trials for DMD.

The citation for this article :

Review of Phase II and Phase III clinical trials for Duchenne Muscular Dystrophy. Scully M, Pandya S, Moxley R. Expert Opinion on Orphan Drugs. Jan 2013; 1(1): 33-46.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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