Preliminary Data from a Phase 1/2 Study of a Gene Therapy for Haemophilia A has been Shared
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Preliminary Data from a Phase 1/2 Study of a Gene Therapy for Haemophilia A has been Shared

Sangamo Therapeutics has shared details of their preliminary data from a clinical trial of the investigational drug SB-525 in patients with Haemophilia A. For more detailed information, you can read…

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Clinical Trial for Duchenne Muscular Dystrophy Gene Therapy Restarts After Hold Lifted

According to a story from statnews.com, the drug developer Sarepta has recently announced the its clinical trial testing an experimental gene therapy has resumed after the FDA lifted the hold.…

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Genetic Condition Observed Only in Mice Discovered in First Patient
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Genetic Condition Observed Only in Mice Discovered in First Patient

According to a report by medicalexpress.com, researchers recently discovered a new genetic condition. Though researchers previously described the condition in animals, no human evidence had yet been found. Michigan State…

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A Group of Disorders Share Similarities in Their DNA 3D Folding Patterns, Researchers Say

Researchers at the University of Pennsylvania have studied the genetics of a class of neurological disorders that includes Huntington’s disease and Fragile X Syndrome. They found that there were similarities…

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A Boy With Congenital Muscular Dystrophy is Being Supported in a Special Way

Michael Dickerson, aged 42, is originally from Hoedspruit Limpopo, South Africa. Recently, Michael was participating in a series of long distance trail runs when he learned about the story of Daniël…

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ICYMI: After Rejection, The Familial Chylomicronemia Foundation Urges The FDA to Consider Approving New Treatment
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ICYMI: After Rejection, The Familial Chylomicronemia Foundation Urges The FDA to Consider Approving New Treatment

According to a story from BioSpace, the U.S. Food and Drug Administration (FDA) recently rejected the approval of the drug Waylivra, which was in development for the treatment of familial…

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