Tag Archives: rare disease

  1. 21st Century Cures Act Passed by House

    Posted on December 1, 201611:03 pm

      Yesterday the House of Representatives passed the draft of the 21st Century Cures Act. It is slated for a vote in the Senate early next week.       The new draft includes: $4.8 billion in new funding for the National Institutes of Health (NIH) $500 million in new funding for the Food and Read more »

  2. Help Support the 21st Century Cures Act and the OPEN ACT

    Posted on November 17, 20164:56 pm

      The final decisions could be made today on the 21st Century Cures Act and the OPEN ACT – a provision that could be transformational for rare disease patients. CureDuchenne signed the letter of support. Despite outreach from constituents, we are still hearing pushback from Sen. Warren on the OPEN ACT. If you could please Read more »

  3. Rare Disease Day

    Posted on February 29, 201611:27 pm

      Today, the last day in February, is Rare Disease Day when the world pauses for a moment to focus on often overlooked diseases that affect relatively few people but in such a devastating way. Among rare diseases Duchenne muscular dystrophy is actually quite rare because it affects less than 20,000 boys in the U.S. Read more »

  4. Update on Phase 3 Tadalafil Trial

    Posted on February 3, 20167:08 pm

    We are sharing the following information from Lilly on their Phase 3 Tadalafil Trial   We are writing to share disappointing news about our Phase 3 study of tadalafil in approximately 330 patients with Duchenne muscular dystrophy (DMD). We recently completed analysis of the initial data from the placebo controlled, double-blind period of the trial, Read more »