Experimental Treatment for Cystic Fibrosis Lung Infections gets Updated Trial Design
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Experimental Treatment for Cystic Fibrosis Lung Infections gets Updated Trial Design

According to a story from BioSpace, the biopharmaceutical company Aridis Pharmaceuticals, Inc., has recently announced that it has struck an agreement with the US Food and Drug Administration (FDA) in…

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September is Charcot-Marie-Tooth Disease Awareness Month: Spreading Rare Disease Awareness
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September is Charcot-Marie-Tooth Disease Awareness Month: Spreading Rare Disease Awareness

September is recognized as Charcot-Marie-Tooth disease (CMT) Awareness Month. Although this illness is one of the most widespread inherited genetic disorders, there is still a significant need to spread awareness…

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New Gene Editing Twist Successfully Treats Myotonic Dystrophy in Mice
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New Gene Editing Twist Successfully Treats Myotonic Dystrophy in Mice

According to an article from ScienceBlog, a twist on CRISPR gene editing has allowed a team of researchers to successfully treat myotonic dystrophy type 1 in mouse models. By modifying…

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Experimental Treatment for Diffuse Intrinsic Pontine Glioma Earns Rare Pediatric Disease Designation
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Experimental Treatment for Diffuse Intrinsic Pontine Glioma Earns Rare Pediatric Disease Designation

According to a story from BioSpace, Ziopharma Oncology, Inc. has recently announced that its investigational therapy Ad-RTS-hIL-12 with veledimex (also called Controlled IL-12) has been given Rare Pediatric Disease designation…

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