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Orphan Disease Remove

January 2018

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By Compliance Global Inc.
This Webinar will discuss the guidelines for marketing authorisation procedures and determinants for successful Orphan Medicinal Product Designation in the EU.
By Cures Within Reach
Batten Disease is a fatal, rare genetic disease affecting the nervous system and most often affecting children. Two families in the Chicago area touched by Batten are making a big difference in research through funds raised by their organizations.
By Cures Within Reach
Jasper Against Batten at Cures Within Reach, a 501(c)3 not-for-profit located in IL, is collaborating with an int'l coalition to fund “Global Gene Transfer for Batten Disease” research at the UNC Gene Therapy Center, announced Dr. Bruce Bloom.
By STATegics, Inc.
STATegics, Inc. awarded a new grant from the Friedreich’s Ataxia Research Alliance (FARA) to advance erythropoietin receptor agonist compounds for the treatment of Friedreich’s ataxia (FRDA).
By Steve Mullen / Fight SMA
Fighting Back, the podcast that tells inspirational stories about people fighting serious or incurable diseases, announces that the latest episode features an interview with the retiring president of the National Organization for Rare Disorders.

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