1. TACT TREAT–NMD Review

    Posted on October 31, 201410:15 pm

      Last weekend, I had the honor, along with Dr. Mike Kelly, CureDuchenne’s Chief Scientific Officer, and other TACT members, to review Duchenne research projects. CureDuchenne was able to look out for the best interest of Duchenne patients by providing a scientific, patient organization and parent perspective.    The projects that we reviewed were: FibroGen Inc, Read more »

  2. Adapting Physical Education for Duchenne Boys

    Posted on October 17, 20144:24 pm

    Those who live with Duchenne muscular dystrophy have physical activity limitations that need to be addressed at school. It is important for Duchenne boys to be included without further damaging their muscles.    Parents need to work with their schools to assist with decision making regarding the most appropriate activities for your student to participate Read more »

  3. Eteplirsen Updates from the World Muscle Society Meeting

    Posted on October 11, 20145:29 pm

      Sarepta provided an 144 week update on eteplirsen at the World Muscle Society meeting. Here are some brief notes. Neutral charge as it passes through kidney; cleared through kidney IV QW Inclusion: >7 years to 13 years (original cohort 7-11 yr) ITT: 12 pts (4 each placebo, 30 & 50 mg/kg) 2 pts stopped Read more »

  4. World Muscle Society Poster Sessions

    Posted on October 10, 20145:32 pm

    WMS Berlin continues to deliver evolving data and novel insights into the treatment, study and diagnosis of Duchenne Muscular Dystrophy.  The location in one of the historical districts for Berlin and German medicine has offered a unique and appropriate setting for keynotes, oral reviews and poster presentations.   Some highlights from the posters over the Read more »

  5. Prosensa begins NDA submission to the FDA for exon-skipping drug drisapersen to treat Duchenne muscular dystrophy

    Posted on 3:38 pm

    CureDuchenne is delighted to share the news that Prosensa announced today that it has begun the submission process for a New Drug Application filing to the FDA for its lead exon-skipping drug candidate, drisapersen, for treating Duchenne. CureDuchenne has supported Prosensa for more than 10 years and we are proud to see this move forward. Read more »

  6. Notes from the World Muscle Society

    Posted on October 8, 20147:31 pm

    The world’s leading experts in the neuromuscular field have gathered in Berlin this week for the World Muscle Society meeting. The focus of the 19th Annual Congress of the World Muscle Society is on the advances in treatment of neuromuscular disorders. Below is a recap of some of the sessions. The morning session, which included Read more »