September 21, 2022 / Community

2022 End Duchenne Tour Recap: Detroit, MI

Parent Project Muscular Dystrophy by: Parent Project Muscular Dystrophy

2022 End Duchenne Tour Recap: Detroit, MI

For the fourth stop of PPMD’s 2022 End Duchenne Tour, we traveled to Detroit, MI. More than 40 families, clinicians, and sponsoring industry partners came together to hear about the latest updates in research, care, and advocacy opportunities for Duchenne and Becker families.

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RESEARCH UPDATES

Research Overview – Eric Camino, PhD (PPMD)

  • Eric presented an overview of the full clinical trial landscape in Duchenne and Becker. He discussed strategies to help your family make decisions about whether to participate in a clinical trial, and how to be fully-informed before enrolling. 
  • PPMD maintains an interactive pipeline on our website that includes all potential treatments being tested with updates from their respective companies, a library of recent webinars, and more.  
  • Download presentation slides and a current list of actively recruiting trials.

Industry Takeaways

  • Edgewise – Abby Bronson, MBA
    • Abby discussed EDG-5506- an oral drug thought to prevent muscle damage by protecting the most susceptible fast muscle fibers. A trial is currently enrolling  adolescent and adult individuals with Becker ages 12+ . An initial study in ambulatory boys is planned to start in the second half of 2022.
    • Download presentation slides
  • NS Pharma – Kathryn Lanza, PhD & Julz Lowenberg
    • Kathryn discussed Viltepso, a weekly infusion for individuals with Duchenne amenable to exon 53 skipping, and ongoing phase 2 and open label extension studies evaluating the safety and efficacy of the drug.
    • Julz spoke about the journey to access of Viltepso, and how NS Support can assist patients and physician offices. You can reach NS support by calling 833-677-8778.
    • Download presentation slides: Viltepso and NS Support
  • Pfizer – Barb Romig, PhD, MBA
    • Barb discussed Pfizer’s Phase 3 Gene therapy trial, CIFFREO, which is looking at both safety and efficacy. This study is currently active and recruiting in both the US and abroad.
    • Slides not available
  • PTC – Malisa Rust, BS, MS
    • Malisa spoke about PTC’s support programs including PTC Cares, a point of contact for patients, providers, and specialty pharmacies. You can reach PTC Cares by calling 1-844-478-2227
    • Download presentation slides
  • Regenexbio – Michelle Gilmor, PhD
    • Michelle discussed RGX-202, an investigational microdystrophin gene therapy, and plans for a phase I/II clinical trial in the U.S. are underway but not yet recruiting.
    • Download presentation slides
  • Sarepta – Hannah Rosenberg, MSc
    • Hannah discussed Sarepta’s 20+ programs in Duchenne. Sarepta has two currently recruiting clinical trials for patients amenable to exon 51 skipping: Mission (eteplirsen PMO) and Momentum (SRP-5051 PPMO). Sarepta is also recruiting for EMBARK, a trial of their investigational gene therapy product, SRP-9001. 
    • Hannah also spoke about SareptAssist, Sarepta’s patient support program to help provide access to therapies. You can reach SareptAssist by calling 1-888-727-3782.
    • Download presentation slides: Sarepta Pipeline and SareptAssist
  • Santhera – Andy Hsieh, PharmD
    • Andy discussed Vamorolone, an investigational drug developed as a dissociative steroid and its recent submission for review by the FDA. 
    • Download presentation slides

Genetics, Your Family, and The Duchenne Registry – Ann Martin, MS, CGC (PPMD)

  • Ann reviewed basic genetics and discussed the Decode Duchenne program which offers free genetic testing and counseling to people with Duchenne and Becker as well as carriers – learn more.
  • Ann also reviewed the importance of participating in The Duchenne Registry and how you can be a citizen scientist by joining and sharing yours or your child’s data.
  • If you have any questions about genetics, testing or clinical trials, please contact one of PPMD’s genetic counselors at 888-520-8675 or coordinator@duchenneregistry.org
  • Download presentation slides

CARE HIGHLIGHTS

We want to thank the following Duchenne specialists for attending this event and providing their expertise to the attendees and answering their questions:

VIRTUAL END DUCHENNE TOUR BAG

CONNECT WITH PPMD’S CARE TEAM 

At PPMD, we place immense value on personal communication with the Duchenne community to provide support and partner with you on this complex journey. PPMD’s Care Team is made up of several staff members with expertise in navigating neuromuscular care and services, and they are available to help answer questions you may have. Schedule a time to meet with PPMD’s Care Team!

SAVE THE DATE

We are so excited to host PPMD’s 2023 Annual Conference, June 29 – July 2, 2023 at The Sheraton Dallas Hotel in Dallas, Texas! Registration will open Spring 2023.

UPCOMING 2022 END DUCHENNE TOUR SCHEDULE:

Be on the lookout this fall for an announcement of the 2023 End Duchenne Tour Stop locations!

*Registration typically opens 6 weeks prior to each event. Visit parentprojectmd.org/tour for more details and make sure you are signed up to receive emails from PPMD to be notified when registration opens. 

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