Loop met Riders for Pelle de Warmathon in Antwerpen

Loop met Riders for Pelle de Warmathon in Antwerpen
Klik op de banner hierboven om in te schrijven voor de Warmathon op 21 december
Deze mountainbiketocht is een initiatief van het Nederlandse Duchenne Parent Project en wordt georganiseerd in samenwerking met de Nederlandse stichting "Evenementen voor Maatschappelijke Organisaties Foundation (Emolife)".

zaterdag 16 oktober 2010

Ataluren Slows the Loss of Walking Ability in Patients with Nonsense Mutation Duchenne/Becker Muscular Dystrophy

Pivotal Data Presented at the World Muscle Society Congress Suggest

Ataluren Slows the Loss of Walking Ability in Patients with Nonsense Mutation Duchenne/Becker Muscular Dystrophy

- 29.7 meter average change in 6-minute walk distance compared to placebo at 48 weeks -
- Safety results show ataluren was generally well tolerated -
- Data to be the basis of interactions with regulatory authorities -

SOUTH PLAINFIELD, NJ – October 15, 2010 – PTC Therapeutics, Inc. today announced that final analyses of Phase 2b efficacy data suggest the investigational new drug ataluren slowed the loss of walking ability in patients with nonsense mutation dystrophinopathy, a disease continuum comprising Duchenne and Becker muscular dystrophy (nmDBMD). These data were presented at the International Congress of the World Muscle Society in Kumamoto, Japan and will be the basis of interactions with the U.S. Food and Drug Administration (FDA) and national regulatory authorities in Europe in the fourth quarter of 2010.