Patient Worthy

4 hours ago

Parent Project Muscular DystrophyPPMD has a long history of working alongside our community to document treatment preferences and share those insights with regulatory authorities such as the FDA. Through town halls, listening sessions, and conversations at community events, we consistently hear that stability is a critically important outcome in both care and treatment.

PPMD has collaborated with researchers at The Ohio State University to conduct a patient preference survey that aims to better understand how individuals with #Duchenne and their caregivers value stability as an outcome of therapeutic intervention, and to capture what stability truly means in the context of treatment and care.

BOTH caregivers and individuals living with Duchenne from the same household are welcome to take the survey! Each person who wants to participate must complete the interest form in order to receive their own individualized survey link.

Check it out: osu.az1.qualtrics.com/jfe/form/SV_d5nXE3fLxqrBxsO ... See MoreSee Less

Patient Worthy

4 hours ago

@OrphanDrug ConferenceFrom June 9-11 in Boston, MA, the World Orphan Drug Congress USA will once again bring together pharmaceutical companies, government officials, patient advocates, and other crucial stakeholders who contribute to developing and improving access to life-saving therapies for rare disease patients. With over 280 speakers and 2,000 attendees, this event is an opportunity to develop innovative solutions to the challenges that orphan drug development poses.

Our Chief Mission Officer, Annie Kennedy, will speak on the first day of the event at 9:20 am during the keynote titled, “Building a Patient-Centered Policy Framework for Rare Disease Drug Development.” The keynote will focus on how policymakers, regulators, and industry can collaborate to create strategies that incorporate patient insights into all stages of drug development.

You can join Annie and the EveryLife Foundation at this event for free with a guest pass. Click the following link to access the guest pass application form to secure a spot: secure.terrapinn.com/V5/sponsor-guest/11031/a0AN200000inZjFMAU?utm_source=partners&utm_medium=eve...

You can learn more about this year’s World Orphan Drug Congress USA here: www.terrapinn.com/conference/world-orphan-drug-congress-usa/index.stm?utm_source=partners&utm_med... ... See MoreSee Less