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Science & Technology

A total of 103 posts are filed under Science & Technology
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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
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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
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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
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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
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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
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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