Rewriting Faulty RNA

When Daniel de Boer’s son was diagnosed with…

Rare Leader: James O’Brien, President of Prader-Willi Syndrome Australia
The Basics Name: James O’Brien Title/Organization: President, Prader-Willi Syndrome Australia; Founding Director, PWS Better Living… Continue Reading
FDA Expands Approved Use of Shire’s Cinryze to Include Prevention of Pediatric HAE Attacks
Rare Daily Staff The U.S. Food and Drug Administration has approved a label expansion for Shire’s C1 esterase inhibitor Cinryze to include… Continue Reading
MHRA Grants MeiraGTx License for London Manufacturing Facility
Rare Daily Staff MeiraGTx The U.K.’s Medicines and Healthcare products Regulatory Agency granted MeiraGTx a Manufacturer’s Authorization… Continue Reading
Alexion Submits Application for Soliris Successor to FDA For Priority Review as a Treatment for PNH
Rare Daily Staff Alexion Pharmaceuticals has submitted an application to the U.S. Food and Drug Administration for the approval of ALXN1210,… Continue Reading
PTC Reports Positive Updated Interim Data for Experimental Drug to Treat SMA Type 1
Rare Daily Staff PTC Therapeutics said updated interim data from the first part of its FIREFISH study investigating its experimental drug… Continue Reading
FDA Removes Clinical Hold on Solid Biosciences Experimental DMD Gene Therapy
Rare Daily Staff The U.S. Food and Drug Administration has lifted the clinical hold on Solid Biosciences’ phase 1/2 clinical trial for its… Continue Reading
New Bluebird Bio Data Shows Gene Therapy Promising for Thalessemia
Rare Daily Staff Bluebird Bio said data from its phase 1/2 Northstar study of its experimental LentiGlobin gene therapy in adolescents and… Continue Reading
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