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Science & Technology

A total of 103 posts are filed under Science & Technology
ArmaGen Announces Initiation of Phase 2 Proof-of-Concept Clinical Trial in Brazil to Study AGT-181 for Treatment of Hurler Syndrome
On March 31st, ArmaGen, Inc., a privately held biotechnology company focused on developing revolutionary therapies to treat severe… Continue Reading
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Unraveling Rare: Mal De Debarqument Syndrome
Mal de Debarqument Syndrome (MdDS) literally means “sickness of disembarkment.” It is a rare and not well understood disorder impacting… Continue Reading
Business Meeting
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
RARE DISEASE REPORT: FDA Approves HAE Drug Berinert For Pediatric Use
The FDA has approved Berinert [C1 Esterase Inhibitor (Human)], to be used in pediatric patients to treat Hereditary Angioedema (HAE)… Continue Reading
Another Big Step Forward For New Cystic Fibrosis Drug By Nivalis Therapeutics
Another big step forward for cystic fibrosis patients! Nivalis Therapeutics, Inc., a clinical stage pharmaceutical company focused on… Continue Reading
After Three Decades, Gene Therapy Comes of Age
By JEFF ST. CLAIR Gene therapy was once viewed as a promising new way to treat many incurable diseases — until a tragic death occurred… Continue Reading