May 18, 2022 / Community

2022 End Duchenne Tour Recap: Atlanta, GA

2022 End Duchenne Tour Recap: Atlanta, GA

The PPMD team was excited to travel to Atlanta for our first in-person End Duchenne Tour stop since 2020. More than 30 people came together to hear about the latest updates in research, care, and advocacy opportunities for Duchenne and Becker families. 



Research Overview – Pat Furlong

Rare Disease Research – Genevieve Wilson

  • Rare Disease Research is an independent research center, located in Atlanta, that specializes in clinical trials for patients with neurogenetic disorders, including Duchenne. Genevieve spoke about the trials currently underway at Rare Disease Research for patients with Duchenne.
  • Download presentation slides

Industry Takeaways

  • Edgewise – Abby Bronson, VP Patient Advocacy
    • Discussed EDG-5506, an oral drug thought to prevent muscle damage  by protecting the most susceptible fast muscle fibers. A trial is being planned in adolescent and adult individuals with Becker ages 12+ to begin in the first half of 2022.
    • Download presentation slides
  • NS Pharma – Julian Mesa, MD, MSL, Medical Affairs & Jonathan Cabral, US Head of Patient Services
    • Julian 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.
    • Jonathan 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
  • Pfizer – Allie Guider
    • Allie announced the FDA has lifted their clinical hold on Pfizer’s investigational mini-dystrophin gene therapy product, fordadistrogene movaparvovec, and plans to open U.S. sites in their phase 3 CIFFREO study by the end of June 2022.
    • Clinical sites have not been announced yet, but questions can be sent to Nicolas Garnier ( at Pfizer.
    • See PPMD’s recent blog post
  • PTC – Audrey Powell, MD, Senior MSL & Patricia Brown, RN, MBA, Patient Engagement Liaison
    • Audrey discussed Emflaza, a corticosteroid approved in the U.S. for use in Duchenne and Ataluren, an investigational product in the U.S. thought to bypass a nonsense mutation to form dystrophin.
    • Patricia 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 – Vivian Fernandez, Executive Director, Patient Advocacy
    • Vivan 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 – Kate Pecora, Senior Specialist, Patient Affairs
    • Kate 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. 
    • Kate 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

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

  • Ann reviewed basic genetics of Duchenne 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 spoke about the importance of participating in The Duchenne Registry and how you can be a citizen scientist by joining and sharing your or your son’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
  • Download presentation slides


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

  • Han Phan, MD, Children’s Hospital Alabama and Rare Disease Research, Atlanta
  • James Poysky, PhD, Katy Child Psychology Associates
  • Meg McKane, MD, Children’s Hospital of Atlanta

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!



We are excited to host PPMD’s 2022 Annual Conference, June 23-26 in Scottsdale, AZ as an in-person and virtual hybrid event this year. Click here to learn more and register.


  • Detroit, MI
  • Boise, ID
  • Indianapolis, IN

*Registration typically opens 6 weeks prior to each event. Visit 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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