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Neuroscience

WMS Poster 2018: JEWELFISH: risdiplam (RG7916) increases SMN protein in patients with SMA that have previously received therapies targeting SMN2 splicing

Oct 03, 2018

JEWELFISH (NCT03032172) is a multicentre, open-label study primarily evaluating the safety and tolerability of once-daily oral administration of risdiplam (RG7916) in patients aged 12–60 years with Type 2 or 3 SMA who have previously participated in a study with therapy targeting SMN2 splicing. Primary endpoints for Part 1 are safety, tolerability and PK/PD of risdiplam.

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Tags: Spinal Muscular Atrophy, Neuroscience, WMS 2018, Poster

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Related Content

Also In WMS 2018:

WMS Poster 2018: SMN protein levels before and after treatment with risdiplam (RG7916) in patients with Type 1 to 3 SMA compared to healthy subjects
Also In WMS 2018:

WMS Poster 2018: SUNFISH Part 1: RG7916 treatment results in a sustained increase of SMN protein levels and the first clinical efficacy results in patients with Type 2 or 3 SMA
Also In WMS 2018:

WMS Poster 2018: A study of risdiplam (RG7916) in babies with pre-symptomatic spinal muscular atrophy

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