“national-organization-for-rare-disorders” Archives

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Entry Author Date Location
Gene Therapy Firms Seek Clarity on FDA Exclusivity, Orphan Designations 09/22/20 National
Arrowhead Pharma Shares Soar on Early Liver Data for RNAi Drug 09/16/20 Wisconsin
J&J Boosts Autoimmune Disease Drug Pipeline With $6.5B Momenta Buyout 08/19/20 New York
RDMD Lands $14M to Expand Reach of Rare Disease Research Platform 04/16/20 San Francisco
AstraZeneca Wins First FDA Drug Nod for NF1, a Rare Genetic Disorder 04/10/20 National
Pfizer Spinout SpringWorks, Migraine Drug Firm Satsuma Prep IPOs 08/16/19 New York
Genmab, Mirum Pharma & Fulcrum Raise $653M in Wall Street Debuts 07/18/19 National
X4 Completes Reverse Merger with Arsanis, Looks Ahead to Phase 3 Test 03/13/19 Boston
Pliant Picks Up $62M to Test Lung, Liver Fibrosis Drug in the Clinic 07/16/18 San Francisco
Teva’s Migraine Drug Fails in Phase 3 for Chronic Cluster Headaches 06/15/18 National
After FDA Approves Bone Disease Drug, Ultragenyx Sets $200K Price 04/17/18 San Francisco
BridgeBio Picks Up Novartis Cancer Drug, Launches New Biotech Startup 01/30/18 San Francisco
Eiger Bio’s Hypertension Drug Flunks Phase 2 Trial, Shares Tumble 01/16/18 San Francisco
NeuroVia Raises $14M to Test Drug for Rare, Fatal Metabolic Disease 07/20/17 Boston
FDA Approves BioMarin Drug, A First for Rare Genetic Batten Disease 04/27/17 San Francisco
With Ovid IPO, Ex-Teva CEO Levin Pitches Neuro Drug Plan to Wall Street 04/11/17 New York
Ionis Pharma Spins Out Lipid Disorder Unit Akcea with Plans for IPO 03/27/17 Boston
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