As CRISPR horizons cloud, a startup focuses on the next big thing with powerhouse potential the RNA editing platform – Endpoints News

Posted: November 11, 2019 at 5:43 pm

Roche has just landed a crucial advance in scoring a come-from-behind win on the spinal muscular atrophy field, giving Novartis and Biogen a run for their money.

The update was brief, but Roche said risdiplam hit the primary endpoint in the placebo-controlled pivotal SUNFISH trial, meeting the threshold for change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment. The results, which is the second, confirmatory portion of a two-part study, involved 180 patients with type 2 or 3 spinal muscular atrophy between 2 and 25 years old.

Unlock this story instantly and join 64,800+ biopharma pros reading Endpoints daily and it's free.

SUBSCRIBE SIGN IN

Go here to read the rest:
As CRISPR horizons cloud, a startup focuses on the next big thing with powerhouse potential the RNA editing platform - Endpoints News

Related Post

Comments are closed.

Archives