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A total of 113 posts are filed under Business
cTAP Announces Two Research Publications Categorizing and Predicting Disease Progression in Duchenne Muscular Dystrophy
The Collaborative Trajectory Analysis Project (cTAP), a public-private partnership to accelerate data science solutions to critical problems… Continue Reading
FDA Approves Vertex’s Orkambi for Cystic Fibrosis Children 6-11 Years Old
by Andrew Black for Rare Disease Report The FDA has approved Vertex Pharmaceuticals’ drug Orkambi (lumacaftor/ivacaftor) for use in… Continue Reading
Color Genomics Raises $45M in Series B. With a Little Help From Bono
Yep, you read that correctly. Maybe the U2 front man has finally found what he’s looking for (sorry) by investing in cancer testing… Continue Reading
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RDR: FDA Approves Cuvitru for Primary Immunodeficiency Disorders
By Andrew Black at Rare Disease Report The FDA has approved Shire’s Cuvitru  [Immune Globulin Subcutaneous (Human), 20% Solution] for… Continue Reading
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Early Stage Investment by CureDuchenne Ventures, LLC Enabled Bamboo Therapeutics to Accelerate Development of a Gene Therapy for Duchenne Muscular Dystrophy
Today, Pfizer Inc. announced its acquisition of Bamboo Therapeutics in a deal worth up to $645 million. Bamboo Therapeutics is an… Continue Reading
Vtesse Secures Support for Product Development and Ongoing Clinical
Vtesse, Inc., a company committed to developing drugs that will benefit patients with extremely rare, life-threatening diseases, announced… Continue Reading
Fierce Biotech: Roivant and Plexcera Launch Rare Disease Focused Spinout
Roivant Sciences and Plexcera Therapeutics have today launched a new biotech company with a singular focus on the ultrarare condition known… Continue Reading