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Innovation

A total of 312 posts are filed under Innovation
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Duchenne Community Rejoices over FDA Approval of Eteplirsen!
CureDuchenne applauds the first-ever approval of a drug in the U.S. to treat Duchenne muscular dystrophy. The drug, Exondys 51… Continue Reading
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Full Behind The Mystery Segment: The Trouble with Diagnosing a Rare Disease
Lifetime’s The Balancing Act premiered it’s newest segment of “Behind the Mystery” earlier this month with an educational story on… Continue Reading
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Can Oxytocin Help Children with Rare Eating Disorder?
by James Radke, PhD Prader-Willi syndrome (PWS) is a genetic disease best characterized by individuals developing an insatiable appetite,… Continue Reading
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ArmaGen Presents Data from First Cohort of Phase 1/2a of AGT-182 for Treatment of Hunter Syndrome
ArmaGen, Inc., a privately held biotechnology company focused on developing revolutionary therapies to treat severe neurological disorders,… Continue Reading
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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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Abeona Therapeutics, EB Research Partnership and EB Medical Research Foundation to Collaborate on Epidermolysis Bullosa (EB) Treatments
Abeona Therapeutics Inc. (Nasdaq: ABEO), a clinical-stage biopharmaceutical company focused on delivering gene and plasma-based therapy for… Continue Reading