Distillery Therapeutics


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<h1 class="text title" doc-editable="text" data-component-id="doc-1ef7l992d0">Repeat expansion-targeting antisense oligo for ALS and frontotemporal dementia</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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

University of Pennsylvania

Mayo Clinic

DISEASE CATEGORY: NeurologyINDICATION:&#xa0;Amyotrophic lateral sclerosis (ALS); frontotemporal dementia......

Neurology

Amyotrophic lateral sclerosis (ALS)

Frontotemporal dementia

Chromosome 9 open reading frame 72 (C9orf72)

Repeat expansion-targeting antisense oligo for ALS and frontotemporal dementia

DISEASE CATEGORY: Neurology<br/>INDICATION:&#xA0;Amyotrophic lateral sclerosis (ALS); frontotemporal dementia Mayo Clinic and Perelman School of Medicine researchers showed that an antisense