Top 7 Most Researched Rare Diseases: Duchenne Muscular Dystrophy

In Advocacy, Clinical Development, Reports by Matt Turner

orphan drug, rare diseases, duchenne muscular dystrophy, sarepta therapeutics, prosensa, shire, pfizer, ptc therapeutics, clinical development, rare disease researchIs there such a thing as low hanging fruit when it comes to rare disease research? While there are fewer than 300 approved orphan drugs for nearly 7000 rare diseases, it is interesting to analyse whether certain rare diseases receive greater attention than others.

Download the DMD Edition of the Top 7 Most Researched Rare Diseases

In the first of a series of eBooks, we will be examining the research and orphan products being developed for Duchenne Muscular Dystrophy, a recessive X-linked form of muscular dystrophy that affects approximately 1 in 3,600 young males and results in muscle degeneration and eventual death. The following pages outlines the 12 organisations with an approved designation for a orphan medicinal product to treat DMD.

In the coming years, it will interesting to see which therapies reach the market first. How will multiple treatments for a single rare disease affect pricing levels? How will each developer demonstrate the value of its orphan drug against another treatment? Will specific therapies target subsets of a rare disease populations?

Importantly, how can the policy makers and key opinion leaders incentivise the rare disease research community to explore possible therapies for continually neglected rare diseases? Do we need a new business model for the most neglected rare diseases? What about a non-profit drug discovery strategy for tackling diseases that show little commercial promise.

Download the DMD Edition of the Top 7 Most Researched Rare Diseases

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Of all the companies developing treatments for Duchenne Muscular Dystrophy, Sarepta Therapeutics, PTC Therapeutics, Pfizer Rare Disease and Shire Rare Diseases are senior sponsors and will be presenting at the World Orphan Drug Congress Europe 2013, 14-15 November 2013, Geneva.