This extra-long mRNA forms toxic clumps in cells, leading to muscle damage and myotonia — when muscles can’t relax after a contraction. Other disease symptoms, such as breathing, heart, and vision ...

Myotonic dystrophy type 2 (DM2), caused by CCTG repeat expansion, is a common adult-onset disorder characterized by myotonia and progressive muscle degeneration. Current therapeutic efforts have ...

Related: Orange Cat Abandoned by Family Still 'Waits at Bus Stop' for Kids to Come Home Trine shared in the video's caption, "We’ve now found out that he has a condition called Myotonia ...

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Myotonia: This is the process in which muscles tense, including both voluntary flexing and involuntary contracting. People can experience an orgasm from stimulation other than in the genital area ...

At this dose, patients experienced correction in a measurement of splicing activity, by an average of 25% on a standard splicing index. DM1 is characterized by myotonia, a symptom marked by muscles ...

SAN DIEGO, April 8, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotid ...

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The trial aims to assess del-desiran's efficacy, with a primary focus on myotonia, a key symptom of DM1. This development is part of Avidity's broader commitment to advancing RNA therapeutics for ...

HARBOR is designed to assess multiple key functional aspects of DM1. The primary endpoint is video hand opening time (vHOT), a measurement of myotonia, which is a hallmark symptom of DM1. Key ...