The neuromuscular group at Great Ormond Street Hospital, London UK, have recently published their study comparing the effectiveness of echocardiogram with cardiac MRI in the assessment Duchenne cardiomyopathy in patients preparing for surgical…

Friday was one of those days, meetings in Washington all week and anxious to get home. Traffic was a mess, Friday evening flights out of DC chaotic. I worried I would miss my flight, the last one to Cincinnati. I attempted to guesstimate the amount of time to get through the security line, grab a salad, and still make the flight. I made it just in time to learn my…

URGE THE SENATE TO PRIORITIZE CURES!

One minute of your day could mean millions of minutes for those diagnosed with Duchenne.

Today, patient advocates from across the country will unite in a day of action to urge the Senate to make medical innovation a top priority.

Earlier this year the Duchenne…

On September 2nd, PPMD convened a public webinar for the Duchenne community focused on FDA Advisory Committee Meetings (or “Ad Comms”). After working closely with representatives from FDA, we are excited to share some of the frequently asked questions that arise regarding the Ad Comm process, as well as some specifically relevant to our community. As we get closer to possible dates for these meetings, we will add information as it becomes available.…

Ad Comms – What’s the buzz all about? If you’ve been anywhere near social media lately, you’ve likely seen the term ‘Ad Comm’ referenced. Speculation about dates, implications for approvals and access to therapies we all care about, questions about who…