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If we are committed to changing the standard of care in rare diseases, we need to see action every single day, not just once a year. 

More than 90 percent of rare diseases have no FDA-approved treatment, and it can take seven to eight years just to get diagnosed. It’s a zero-sum game for marketers because we’re walking onto an empty field full of people desperate for someone to join their team. We have the opportunity to make a substantial impact in the small towns of rare disease.

Almost a month ahead of schedule, the U.S. Food and Drug Administration has approved Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy in adults.

It’s game day for bluebird bio. The company, battered by layoffs and cash concerns, faces a two-day crucible as the U.S. Food and Drug Administration’s Cell, Tissue and Gene Therapies Advisory Committee will give two lentiviral vector gene therapies a thumbs up or down.

Vertex Pharmaceuticals announced on Wednesday that the U.S. Food and Drug Administration granted Breakthrough Therapy Designation to the company’s candidate, inaxaplin (VX-147). Inaxaplin was granted the designation for the treatment of APOL1-mediated focal segment glomerulosclerosis (FSGS).

Bluebird bio’s blood disorder treatment demonstrates “clinically meaningful” benefit in patients, staff reviewers at the U.S. Food and Drug Administration said in briefing documents published on Tuesday.

Dublin, Ireland– April 30, 2015 – Shire plc (LSE: SHP, NASDAQ: SHPG) announces the appointment of Jeff Poulton as Chief Financial Officer (CFO) and member of the Executive Committee. Jeff will additionally join the Shire Board of Directors. Both appointments are effective immediately.  Jeff has served as Interim CFO since December 2014, while overseeing Investor […]