STUDY: Pitolisant Reduced Fatigue and Daytime Sleepiness in Adults with DM1
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STUDY: Pitolisant Reduced Fatigue and Daytime Sleepiness in Adults with DM1

An estimated 80% or more of people with myotonic dystrophy type 1 (DM1) struggle with severe fatigue and excessive daytime sleepiness (EDS). While most people consider myotonia (inability to contract…

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AOC 1001 Earns Breakthrough Therapy Designation for Myotonic Dystrophy Type 1 (DM1)
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AOC 1001 Earns Breakthrough Therapy Designation for Myotonic Dystrophy Type 1 (DM1)

Currently, there are no approved treatment options for myotonic dystrophy type 1 (DM1), a progressive neuromuscular disease. As the disease progresses, affected individuals experience muscle weakness, respiratory distress, and cardiac…

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How PepGen’s Jane Larkindale and Alayna Tress Advocate for Patient-Centricity in Rare Disease Drug Development
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How PepGen’s Jane Larkindale and Alayna Tress Advocate for Patient-Centricity in Rare Disease Drug Development

Contributed by Jane Larkindale and Alayna Tress While millions of people globally are living with a rare disease, patients often find it difficult to feel seen or heard throughout their…

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The Link Between Myotonic Dystrophy Type 2 and Recurrent Ventricular Tachycardia Needs More Research

Myotonic dystrophy type 2 is a rare and poorly understood condition. Even less understood is its relation to recurrent ventricular tachycardia, which is even rarer. Myotonic Dystrophy Type 2 Myotonic…

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Study Shows How to Better Predict Pacemaker Needs in Myotonic Dystrophy Patients

A recent study has examined what risk factors exist for patients diagnosed with myotonic dystrophy, the most common of all muscular dystrophies. This is a progressive, hereditary condition. Regarding cardiac…

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Avidity Announces 2021 Pipeline Updates For DM1, FSHD, DMD
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Avidity Announces 2021 Pipeline Updates For DM1, FSHD, DMD

In early January 2021, biopharmaceutical company Avidity Biosciences ("Avidity") announced updates from its 2021 pipeline. Currently, Avidity is working to create a line of Antibody Oligonucleotide Conjugates (AOCs), therapeutic options…

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AMO-02, a Congenital Myotonic Dystrophy Treatment, Granted Rare Pediatric Disease Designation
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AMO-02, a Congenital Myotonic Dystrophy Treatment, Granted Rare Pediatric Disease Designation

The FDA has recently granted the Rare Pediatric Disease (RPD) designation to AMO-02, a treatment for congenital myotonic dystrophy. As there is currently an unmet medical need for this condition,…

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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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