Distillery Therapeutics


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<h1 class="text title" doc-editable="text" data-component-id="doc-1ef7l992d0">Scripps team discovers RNA-targeting small molecule for myotonic and corneal dystrophies</h1><div class="byline ng-tns-c17-4 ng-star-inserted" data-component-id="doc-1ef7l9he70">
    <span class="ng-tns-c17-4" doc-editable="text">By Claire Quang, Staff Writer</span>
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

Scripps Research

DISEASE CATEGORY: Musculoskeletal; ophthalmic diseaseINDICATION:  Myotonic dystrophy type 1 (DM1); ophthalmic......

Musculoskeletal

Ophthalmic

Myotonic dystrophy type 1 (DM1)

Ophthalmic (unspecified)

Muscleblind-like splicing regulator 1 (MBNL1)

Scripps team discovers RNA-targeting small molecule for myotonic and corneal dystrophies

DISEASE CATEGORY: Musculoskeletal; ophthalmic disease<br/>INDICATION: Myotonic dystrophy type 1 (DM1); ophthalmic A Scripps Research team led by Expansion Therapeutics Inc. co-founder Matthew Disney has