welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- login
Guides & Guidelines
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene
source: National Institute for Health and Care Excellence
year: 2016
summary/abstract:This guidance is not intended to affect the position of patients whose treatment with ataluren was started within the NHS before this guidance was published. Treatment of those patients may continue without change to whatever funding arrangements were in place for them before this guidance was published until they and their NHS clinician consider it appropriate to stop.
read morerareRelated
-
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who H...This study is designed to generate addit...
-
Ataluren treatment of patients with nonsense mutation dystrophinopathyIntroduction: Dystrophinopathy is a ra...
-
Boy with muscular dystrophy writes heartfelt note to Scottish leaderMichael Young, 9, wrote a letter to Scot...
-
Study of Ataluren (PTC124®) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dyst...Duchenne/Becker muscular dystrophy (DMD/...
-
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)Duchenne muscular dystrophy (DMD) is a g...
-
Safety and Tolerability of Ataluren in a Phase 3 Study of Patients with Nonsense Mutation Duchenne Muscular Dystroph...Objective: Examine the safety/tolerabil...
-
Clinical trial shows some promise for Duchenne muscular dystrophy drugThe results of an international clinical...
-
PTC Therapeutics Announces Initial Data from Patient Registry Demonstrating Translarna™ (ataluren) Slows Disease P...PTC Therapeutics, Inc. today announced p...
