| Ultragenyx Pharma Wins FDA’s First Drug Nod for Rare Metabolic Disorder | Frank Vinluan | 06/30/20 | San Francisco |
| In an About-Face, FDA Approves Second Sarepta Muscular Dystrophy Drug | Sarah de Crescenzo | 12/13/19 | Boston |
| Astellas Bets On Gene Therapy With $3B Audentes Acquisition Offer | Sarah de Crescenzo | 12/03/19 | National |
| After AGTC Deal Flops, Biogen Bets $800M on Gene Therapy Rival Nightstar | Ben Fidler | 03/04/19 | Boston |
| With Its First Human Data, Solid Slips in Duchenne Gene Therapy Race | Ben Fidler | 02/07/19 | Boston |
| FDA Halts Clinical Test of Zafgen Diabetes Drug, Stock Price Tumbles | Frank Vinluan | 11/26/18 | Boston |
| Data Starts Stretch Run for UniQure’s Upgraded Hemophilia Gene Therapy | Ben Fidler | 11/15/18 | Boston |
| Vertex Hums Along as AbbVie Snatches Up a Rival’s CF Drugs for $45M | Ben Fidler | 10/25/18 | Boston |
| FDA, Following EMA, Nixes Amicus’ Plans for Speedy OK of Pompe Drug | Ben Fidler | 09/10/18 | New York |
| Ultragenyx Makes Competing Bid for Gene Therapy Biotech Dimension Tx | Frank Vinluan | 09/18/17 | San Francisco |
| Amicus’s Scioderm Deal Flops as Skin Drug Fails in Phase 3 | Ben Fidler | 09/13/17 | New York |
| Ophthotech Eye Drug Fails Badly in Pivotal Test, Shares Crumble | Ben Fidler | 12/12/16 | New York |
| On Front Lines Of Price Dispute, Eye Doctors Wary of New Drug Data | Ben Fidler | 11/01/16 | National |
| Bio Execs Talk Patient Advocacy, Duchenne Approval at BioForward Panel | Jeff Buchanan | 09/28/16 | Wisconsin |
| Sarepta Prices $300K Duchenne Drug As FDA Rift Emerges Over Approval | Ben Fidler | 09/19/16 | Boston |
| At Duchenne Panel, Parents Plead With Experts, FDA Decision Looms | Ben Fidler | 11/24/15 | San Francisco |
| Biogen Drug Shows Early Promise in Slowing Alzheimer’s Decline | Alex Lash | 03/20/15 | Boston |
| Amicus Counts on “Chaperone” Tech to Enhance Rare Disease Treatments | Arlene Weintraub | 01/11/12 | New York |
