Protein replacement therapies for rare diseases: a breeze for regulatory approval?
نویسندگان
چکیده
Protein replacement therapies for rare monogenic diseases have a higher probability of regulatory approval compared with biologics, small molecules, and grant-funded orphan drugs.
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عنوان ژورنال:
- Science translational medicine
دوره 5 178 شماره
صفحات -
تاریخ انتشار 2013