1. Thoughts about PTC

    Posted on July 25, 20165:32 pm

    The announcement that PTC recently submitted an appeal to elevate the “Refusal to File” of Translarna (also known as ataluren and PTC124) to the next level of FDA management is an opportunity for the Duchenne community to raise their voices and let their experiences and opinions be heard! Translarna is the only therapy under investigation Read more »

  2. Summer Road Trip

    Posted on July 13, 20164:49 pm

      Guest blog by Hawken Miller Many of the things I don’t think are possible for me to do really are. Once I get over my initial apprehension and thoughts about what I won’t be able to do and I open my mind I find that anything I set my mind to is possible. This Read more »

  3. BioMarin Update for the Duchenne Community: 7th July, 2016

    Posted on July 7, 20163:00 pm

      BioMarin is providing a further update to the Duchenne Community following our announcement of the withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on compounds BMN 044, BMN 045 and BMN 053. Update for Patients Receiving Drisapersen, BMN 044, 045 or 053 in Clinical Read more »