JEWELFISH

November 1, 2018November 5, 2018

Risdiplam Showing Potential to Raise SMN Protein to Healthy Levels in SMA Babies and Adults, PTC Says in Interview

News

Preliminary data from two ongoing clinical trials into risdiplam (RG7916) — a potential oral therapy for all types of spinal muscular atrophy — support its considerable promise, with treated babies ... Read more

June 6, 2018June 6, 2018

Roche Stops Work on Olesoxime After Disappointing Long-term Results in Phase 2 Trial

News

Roche has stopped the development of olesoxime, a treatment candidate for spinal muscular atrophy (SMA) following disappointing 18-month results in an ongoing clinical study, the company announced. In a statement ... Read more

April 25, 2018June 18, 2018

#AAN2018 — RG7916 Works to Raise SMN Protein Levels in Babies with SMA Type 1, Trial Shows

News

A potential oral treatment for children with type 1 spinal muscular atrophy (SMA) called RG7916 was able to safely raise levels of the crucial SMN protein that is lacking in these patients, ... Read more

April 18, 2018April 27, 2018

#AAN2018 – SMA Treatment Candidate RG7916 Increases Amount of Key SMN Protein, Clinical Trials Show

News

Roche’s treatment candidate RG7916 increased the amount of full-length, functional SMN protein in spinal muscular atrophy (SMA) patients, according to soon-to-be-presented data from a Phase 2/3 clinical trial. The company will ... Read more

February 2, 2018

RG7916 Safe for Infants With Type 1 SMA, Early Data Indicate

News

Early interim data from a Phase 2 clinical trial evaluating varying doses of a potential oral therapy, RG7916, in infants with type 1 spinal muscular atrophy (SMA) is showing ... Read more

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