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Neuroscience

Preliminary results from a 15-month open-label extension (OLE) study investigating RG6042 huntingtin protein (HTT) antisense oligonucleotide (ASO) in adults with manifest Huntington’s disease (HD)

Feb 27, 2020 by [Schobel SA]

Long-term safety, tolerability and biomarker effects of tominersen (most recently known as RG6042) were evaluated in participants of the 15-month open-label extension of the Phase I/IIa study. Results of this preliminary 15-month analysis will be presented.

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Tags: Oral Presentations, Huntington's Disease, Neuroscience, CHDI 2020

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Also In Huntington's Disease:

Preliminary results from a 15-month open-label extension (OLE) study investigating RG6042 huntingtin protein (HTT) antisense oligonucleotide (ASO) in adults with manifest Huntington’s disease (HD)
Also In Oral Presentations:

AAN Presentation 2019: Update from SUNFISH part 1: Safety, tolerability and PK/PD from the dose-finding study, including exploratory efficacy data in patients with Type 2 or 3 spinal muscular atrophy (SMA) treated with risdiplam (RG7916)
Also In Oral Presentations:

FIREFISH Part 2: Efficacy and safety of risdiplam (RG7916) in infants with Type 1 spinal muscular atrophy (SMA)

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