Myotic Dystrophy Treatment Breakthrough

PLUS: Mysteries Surround Ehlers-Danlos Syndrome

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  • Mysteries Surround Ehlers-Danlos Syndrome READER REQUEST

  • Measles to Kill Medulloblastoma READER REQUEST

  • Let There Be Light: Gene Therapy for Blindness

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✋🏼🤚🏼 Myotonic Dystrophy Treatment Breakthrough

Congenital Myotonic Dystrophy (CMD) is an inherited genetic disorder characterized by progressive muscle wasting and weakness, among other symptoms. For example, imagine opening a door and having difficulty releasing your grip on the doorknob.

1/ CMD is caused by a mutated DMPK gene. Specifically, sections of the DMPK gene are incorrectly repeated thousands of times. As a result, the abnormally large gene produces an unusually large mRNA, producing a nonfunctional protein that causes the disease.

2/ Avidity Biosciences has developed AOC 1001, designed to address CMD's root cause.

Here’s how AOC 1001 works👇🏼

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