Blog

August 12, 2024

PPMD Urges FDA to Conduct a Full Review of Ataluren (Translarna) Following Resubmission of NDA

PTC Therapeutics has shared that it has resubmitted its New Drug Application (NDA) to the FDA for review and potential approval of ataluren (Translarna). Ataluren is an oral small molecule treatment that allows for stop-codon…

Learn More

Clinical Trials & Outcome Measures in Duchenne

Clinical trials and research studies are vitally important to improving health and quality of life for people with Duchenne and Becker. Research is always advancing and evolving, and outcome measures fall into this category, as…

Learn More

Making it Work: Practical Tips for Enhancing Quality of Life

It’s important to ensure that even within the context of Duchenne/Becker, kids are doing things that kids should be doing, adults are doing things that adults should be doing, and families can lead the lives…

Learn More

ITF Therapeutics Announces U.S. Commercial Availability of DUVYZAT™ (givinostat) for Treatment of Duchenne

PPMD is pleased to learn that DUVYZAT™ (givinostat) is now commercially available in the United States. DUVYZAT™ is a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne from six years of age and…

Learn More

Navigating the Approved Therapy Landscape

The approved therapy landscape has changed drastically in Duchenne, especially over the last year. With multiple newly approved therapies comes a lot of questions about how to navigate these drugs: when they are prescribed; who…

Learn More

PPMD Convenes Leading Experts to Discuss Topics in Becker Care Ahead of 30th Annual Conference

Leading into PPMD’s 30th Annual Conference, PPMD is excited to host a professional meeting focusing on Becker muscular dystrophy care. Over the course of two days, attendees from across subspecialties, academic medical centers, and industry…

Learn More

FDA Expands ELEVIDYS Label Indication for Ages 4 Years and Older

PPMD is excited to share that the FDA has made the decision to expand the labeled indication for ELEVIDYS, a micro-dystrophin gene therapy used to treat people with Duchenne muscular dystrophy, to include individuals at…

Learn More

PAAC Travel Tips

Anyone living with Duchenne muscular dystrophy knows that there are extra steps to EVERYTHING. Traveling with medical equipment requires planning and organization. PPMD’s Adult Advisory Committee (PAAC) has put together several resources with tips that…

Learn More

Newborn Screening for Duchenne: PPMD’s Collaborative Future Planning Efforts

On Saturday, June 8th, PPMD convened a vital meeting of physicians, physical therapists, genetic counselors, data scientists, and other key opinion leaders to discuss the development of data infrastructure and care planning for newborns that…

Learn More

Cranbury Pharmaceuticals (Tris Pharma) Receives FDA Approval for First Generic Version of Emflaza® (deflazacort) for Duchenne

Cranbury Pharmaceuticals, a subsidiary of Tris Pharma, today announced the U.S. Food and Drug Administration (FDA) approved the Abbreviated New Drug Application (ANDA) for the first generic version of Emflaza® oral suspension (deflazacort) for the…

Learn More

Join Our Mailing List