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Entry Author Date Location
Breaking Bottlenecks, Opening the Floodgates to Genomic Data 11/18/14 National
With $22M, True North Points Toward Rare-Disease Clinical Trials 06/17/14 San Francisco
LTI Corrals $5M to Exploit Link Between Gaucher, Parkinson’s 05/12/14 Boston
Repligen, Continuing Overhaul, Flips Rare Disease Drugs to BioMarin 01/21/14 Boston
Moderna, Living a Charmed Life, Pulls in Another $125M From Alexion 01/13/14 Boston
Gates Foundation Health Chief on Coaxing Pharma to Do Good 11/25/13 National
NEA, Genzyme Join $18M Round For Rare Disease Startup Edimer 07/30/13 Boston
Aegerion Shares Soar on New Numbers For Rare Disease Drug 07/30/13 Boston
Sarepta to Seek FDA Approval For Duchenne Muscular Dystrophy Drug 07/24/13 Boston
Ensemble Adds to Cast of Partners With Alexion Deal 07/18/13 Boston
Alnylam Shares Boom on Early Data For Subcutaneous RNA Drug 07/11/13 Boston
Alnylam’s Amyloidosis Drug Holds up in Second Clinical Trial 07/01/13 Boston
Amicus Shelves New-Drug Filing For Fabry Disease Drug 06/17/13 New York
Profiles in Long-Termism: Sarepta Therapeutics CEO Chris Garabedian 06/10/13 National
Nimbus Discovery, Shire to Co-Develop Oral Rare Disease Drugs 05/08/13 Boston
Repligen in Drug Development Licensing Deal with Pfizer 01/04/13 Boston
Synageva BioPharma Announces $80 Million Stock Offering 01/03/13 Boston
Amicus-GlaxoSmithkline Drug for Fabry Disease Fails Clinical Trial 12/20/12 New York
Promedior Joins Ranks of Woman-Helmed Biotechs in Massachusetts 12/19/12 Boston
East Coast Life Sciences Roundup: Insider Trading, Shire, Pfizer 11/23/12 Boston
Alnylam Gets $22.5M From Genzyme for Asia Rights to Amyloidosis Drug 10/22/12 Boston
Rare Disease Science Challenge Offers $400,000 in Cash & Services 10/15/12 San Diego
Sarepta Moves From Seattle to Boston for the Talent 09/07/12 Seattle
Two Years After Rising from Ashes, Aegerion Preps for Drug Debut 08/09/12 Boston
NPS Rallies as Good News Flows for Two Rare-Disease Drugs 06/25/12 New York
Ra Pharma Pours $8.6M Into Discovery Tech and New Rare-Disease Drug 05/23/12 Boston
Pfizer Takes on Genzyme With New Gaucher Drug 05/02/12 Boston
Hard-Nosed Hedge Fund Chief Has Soft Spot for Rare-Disease Startup 04/03/12 New York
Amicus Counts on “Chaperone” Tech to Enhance Rare Disease Treatments 01/11/12 New York
Sanofi Names David Meeker New CEO of Genzyme, Will Report to Viehbacher 10/24/11 Boston
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