Share 'Industry partners receive FDA’s Pediatric Rare Disease Designation – and YOU helped make it possible!'
In the last few weeks we’ve heard the news from several companies working to develop therapies for Duchenne that they have received Rare Pediatric Disease Designations from the FDA. Santhera, Sarepta, Catabasis, and Biomarin.
We as a community have celebrated this news. But what does it actually mean?
And did you know that it would not have been possible without YOUR tireless advocacy and t…
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