Four drugs are vying to be 1st approved for treating patients with Duchenne muscular dystrophy (DMD). Of the 4 drugs in late stage development, 3 will only be effective for subsets of the DMD population (10-15%) and 2 of those 3 are for the same subset (exon 51 mutation). And the 4th drug is being developed to treat all DMD patients but will probably only slow progression of the disease slightly. The other 3 will hopefully deter progression of the disease much longer. But that is just speculation. We still need to see the phase 3 data for all of them.
So which drug will be the first? – See more at: http://www.raredr.com/front-page-legislation/articles/which-orphan-drug-will-be-first-approved-duchenne-muscular-dystrop-0#sthash.d2EobOGg.dpuf
The speaker faculty for the 4th Annual World Orphan Drug Congress is being finalised this month and so far there has been unprecedented interest and endorsement from the global biopharma and rare disease industry. Take a look at the sponsoring organisations here and book today to get involved with the leading figures of the orphan drug industry, expand your rare disease network and share ideas to create new solutions to meet your business objectives in this space.