Experimental Gene Therapy for Type 1 Spinal Muscular Atrophy Gets Priority Review

According to a story from BioPortfolio, the drug developer Novartis recently announced that the US Food and Drug Administration (FDA) has granted the company's investigational gene therapy AVXS-101, also known…

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Potential Dravet Syndrome Treatment Shows Capability in Clinical Trial
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Potential Dravet Syndrome Treatment Shows Capability in Clinical Trial

According to a story from pm360online.com, the pharmaceutical company Zogenix Inc. recently announced that its experimental drug candidate ZX008, also known as Fintepla®, has performed well in the company's latest…

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Is This Combination Treatment The Key to Treating Digestive Tract Cancers?
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Is This Combination Treatment The Key to Treating Digestive Tract Cancers?

According to a story from EurekAlert!, the results from a recent Phase 2 clinical trial suggest that a two drug combination could be a useful approach for people with rare,…

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Teen Overcomes Rare Disease That Causes Severe Shoulder Pain to Dance in the Nutcracker 
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Teen Overcomes Rare Disease That Causes Severe Shoulder Pain to Dance in the Nutcracker 

Behold the power of sheer will, determination, and tenacity! New York teen Hannea Milliman overcame the rare disorder Parsonage Turner Syndrome - which causes severe pain in the shoulder and arm -…

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Paris Parents Start “Kisses for Leo” Campaign for son With Rare Infantile Neuroaxonal Dystrophy
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Paris Parents Start “Kisses for Leo” Campaign for son With Rare Infantile Neuroaxonal Dystrophy

  When Deborah, a New Yorker, flew over to Paris to do an internship for an architecture office, she never knew she would be flying across the world and meeting…

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Experimental Gene Therapy for Fanconi Anemia Earns Fast Track and Regenerative Medicine Advanced Therapy Designation

According to a story from BioPortfolio, the gene therapy company Rocket Pharmaceuticals has recently announced that the company's investigational gene therapy RP-L102 has earned both Fast Track and Regenerative Medicine…

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