Revised Duchenne Anesthesia Recommendations
New evidence suggests that people with Duchenne are not at an increased risk of developing malignant hyperthermia, but may continue to be at an increased risk of rhabdomyolysis (serious and sometimes fatal muscle breakdown) when administered inhaled anesthetic gases. In light of this new knowledge, PPMD has joined with experts in muscle disease and anesthesia to clarify our guidelines on the administration of general anesthesia for patients with Duchenne.
Webinar Recording: PF-06252616 (Anti-Myostatin)
On June 3, 2015, Dr. Michael Binks, Vice President for Rare Disease Clinical Research at Pfizer, presented information about the upcoming clinical trial of PF-06252616. The webinar provides an overview of Pfizer’s approach to advancing the science of an anti-myostatin monoclonal antibody as an investigational compound for potential therapeutic use in muscle wasting diseases, such as Duchenne muscular dystrophy.
Live from PPMD's Connect Conference! Urge your Senators to Cosponsor PFIA (S. 1597) from Home!
Yesterday Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) introduced S. 1597, the Patient-Focused Impact Assessment Act, which seeks to drive FDA use of patient focused drug development tools. We need you to reach out to your Senators today and urge that they sign on as a cosponsor of S. 1597.
PPMD's Annual Connect Conference: Streaming Schedule
Can't make it to PPMD's 2015 Annual Connect Conference? We will be live streaming most sessions Thursday, June 18 - Saturday evening, June 20.
End Duchenne eNews: Providing Guidance, Providing Strength
Catch up on the latest research and community updates in this month's End Duchenne eNews!
PPMD Names Duke Medicine Certified Duchenne Care Center
PPMD is excited to name Duke Children’s the ninth Certified Duchenne Care Center! PPMD recognizes the development of Duke Children’s multidisciplinary care team and their dedication to providing the best care and services for people and families living with Duchenne.
Breaking News! FDA Releases Draft Guidance for Duchenne
Today the FDA has released their draft guidance on Duchenne. This is the first time that a patient community has proposed and drafted a guidance for FDA to use as a jumping off point for their own drug development guidance an official map for how companies can move their Duchenne-focused innovation through the regulatory approval process.
Update on the FDA Guidance
Last week we met with FDA to discuss status and potential release of guidance to help accelerate development and review of potential therapies for Duchenne. PPMD President Pat Furlong provides an update from the meeting.
September 4-6, 2015 in Disneyland (California)