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Archives for: August 9th, 2018

2017 Rare Diseases and Orphan Products Breakthrough Summit

Written by Christina Jensen on October 16, 2017

RareSummit_Logo_RGB_smallNORD’s Rare Diseases and Orphan Products Breakthrough Summit is the largest and most meaningful multi-stakeholder event of its kind – historically featuring over 20 speakers from the FDA, participation from over 80 patient organizations and the Pharma/Biotech industry’s foremost experts in orphan product innovation,… Read More

FDA Announces First Gene Therapy in U.S.

Written by Mary Dunkle on August 31, 2017

In an action the rare disease community has been eagerly awaiting, the Food and Drug Administration (FDA) has announced the first-ever approval of a gene therapy in the U.S. The product is Kymriah, and it is approved to treat certain children and young adults who have a cancer of blood… Read More