Myotonic Dystrophy Type 1: RNA-Targeted CRISPR Shows Promise in Mouse Model
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Myotonic Dystrophy Type 1: RNA-Targeted CRISPR Shows Promise in Mouse Model

According to a story from PR Newswire, the RNA-targeted gene therapy company Locanabio, Inc. has recently released findings from a preclinical evaluation of an RNA-targeted CRISPR Cas9 (RCas9) system as…

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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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Triplet Therapeutics Will Participate in END-DM1 for Myotonic Dystrophy

Last week, biotechnology company Triplet Therapeutics announced their participation in END-DM1, a natural history study to learn more about myotonic dystrophy. There are two major forms of myotonic dystrophy, a rare…

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Using PacBio SMRT Sequencing in Myotonic Dystrophy Type 1 and Other Rare Disorders

  Stéphanie Tomé is an investigator at the Sorbonne Université in Paris, France. A recent article in PacBio describes Tomé’s research into a disease that becomes progressively worse with each generation.…

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Gene Therapy for 12 Rare Diseases will be Investigated Thanks to this Industry-Academia Partnership

Extended Collaboration  Amicus Therapeutics has just announced that they are expanding their collaboration with the University of Pennsylvania's Perelman School of Medicine for the next five years. This collaboration is…

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AMO Pharma Presents New Rating Scale for Type 1 Myotonic Dystrophy Symptoms
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AMO Pharma Presents New Rating Scale for Type 1 Myotonic Dystrophy Symptoms

A press release from American biopharmaceutical company AMO Pharma, published by PR Newswire, detailed the recent completion of the Company's new congenital myotonic dystrophy type 1 (CDM1) symptom-evaluation scale. The…

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Company Announces Plans for Myotonic Dystrophy and Duchenne Muscular Dystrophy Treatments
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Company Announces Plans for Myotonic Dystrophy and Duchenne Muscular Dystrophy Treatments

According to a story from CRWE World, the genetic medicines company Audentes Therapeutics has recently announced its intent to develop new therapies for myotonic dystrophy and Duchenne muscular dystrophy. These…

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Challenges and Benefits: A Family’s Experience at the Myotonic Dystrophy Consortium
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Challenges and Benefits: A Family’s Experience at the Myotonic Dystrophy Consortium

The International Myotonic Dystrophy Consortium was held in San Francisco last month and while Patient Worthy was not there in person we were fortunate to have a family reporting from the…

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