linkedin post 2013-03-14 16:58:46

A GREAT SUMMARY OF ISSUES IN RARE DISEASE DRUGS. A hot trend, initiated long ago by Genzyme in particular, with a number of players including BioMarin, and today even Big Pharma appears interested in this field. In some cases, the diseases are well defined (leukodystrophies) at a genetic level, so their patient pools are homogeneous; knowing the genetic lesion enables pathway targeting. Increasingly, patient organizations fund clinical trials and bring well characterized patient registries to the table.

http://lnkd.in/NYbTxH

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