February 14, 2024

WATCH: PTC Therapeutics — EMFLAZA® Landscape in 2024 (Webinar Recording)

PPMD was recently joined by the team at PTC Therapeutics to discuss upcoming changes and continuing community support around EMFLAZA (deflazacort). EMFLAZA came on the U.S. market in 2017 as the first FDA-approved steroid specifically…

Learn More

February 9, 2024

Update From PPMD’s 2024 PPMD Duchenne Healthcare Professionals Summit

PPMD recently hosted our fifth in-person Duchenne Healthcare Professionals Summit in Sanibel, FL. Over the course of three impactful days, we were joined by around 300 healthcare professionals, researchers, and industry partners to collaborate, connect,…

Learn More

January 30, 2024

Watch: Navigating Steroid Choices – Understanding the Process, Pathways, and Policies (Webinar Recording)

Last week, PPMD was joined by Kathy Mathews, MD (Neurologist), Christina Trout, RN, MSN (Clinic Coordinator), and Rachel Kinn, PharmD, BCPPS (Pharmacist) from the University of Iowa to discuss decision making regarding steroids. In recent…

Learn More

January 29, 2024

PPMD 2023 Duchenne Early Care Meeting Report Published: Enhancing Support in Early Diagnosis

Last year, PPMD hosted a Duchenne Early Care meeting as part of the 2023 Duchenne Healthcare Professionals Summit, focusing on key aspects of care for the youngest children in our Duchenne community, in preparation for…

Learn More

January 26, 2024

Duchenne Added to Minnesota’s Newborn Screening Panel

PPMD is excited to announce another significant milestone: Minnesota has officially approved the addition of Duchenne to the state’s newborn screening panel! Approximately 63,000 babies are born in Minnesota every year, which means that we…

Learn More

January 25, 2024

CHMP Issues Negative Opinion for Renewal of Conditional Marketing Authorization for Translarna™ (ataluren) in Europe

PPMD is disappointed to learn that the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) today issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarna™…

Learn More

January 17, 2024

PPMD Hosts 2024 Duchenne Healthcare Professionals Summit

Each January, PPMD brings together experts in the field for our Duchenne Healthcare Professionals Summit to ensure that caring for individuals with the latest information and technology remains a constant priority.  Healthcare professionals from PPMD’s…

Learn More

January 11, 2024

Introducing PPMD’s Gene Therapy Hub

We are excited to announce the launch of PPMD’s Gene Therapy Hub! Gene therapy has made significant strides in Duchenne, with one therapy approved and other potential therapies in various stages of development. As our…

Learn More

January 5, 2024

The Importance of Assisted Cough

It’s winter, which means respiratory virus season is upon us. Those living with Duchenne and Becker may find themselves needing more support to help clear thick secretions that often accompany respiratory illness. Using assisted cough…

Learn More

December 20, 2023

CDCC Spotlight: Riley Hospital for Children

The Little Things There are many ways the team at Riley Children’s Certified Duchenne Care Center go the extra mile. One such way is by providing patients with individualized toys and activities for each of…

Learn More