September 14, 2020 / Advocacy,Care,Clinical Trials,Community,Research

Resilience Is Strength: Thank You for Fueling Our Fight to End Duchenne

As we celebrate the resiliency of our community this week as part of Duchenne Action Month, I have been reflecting upon all that we continue to accomplish, even in this time that is anything but normal. It is you that keeps us going, and I am forever grateful.

Even in these past few months, feeling upside-down most of the time, PPMD has continued onward no matter the obstacles in our way. I have seen dedication, flexibility, creativity, compassion, and perseverance from our partners and the PPMD team, and I know we will emerge even stronger when we see the other side of this time.

Connecting the Community

One of my proudest moments this summer was PPMD’s Virtual Annual Conference. This year’s Conference changed the way we can connect as a community, allowing us the latest updates on clinical trials and care, while reaching more families than ever before. Looking towards 2021 and beyond, we are already making plans to ensure future Conferences blend hybrid and in-person options to increase the reach of this one-of-a-kind event. I hope you will spend time on our Conference Hub as it is full of resources and full recordings of the event.

Continuing Our Pandemic Response

In response to COVID-19, PPMD released a survey earlier this spring to better understand the patient experience with care, trials, and access to approved therapies during the pandemic.  We recently released the results of the survey, which included key learnings such as the impact of COVID- 19 on mental health. Thanks to your contributions we have been able to share data-driven information with members of our Duchenne Drug Development Roundtable as they address the challenges imposed by the pandemic. PPMD has also been working closely with other rare disease organizations and recently joined dozens of patient organizations in a letter to Congress urging them to protect immunocompromised patients during the pandemic.

Advancing The Duchenne Registry

Our Duchenne Registry team continues to provide resources to families and improve the largest global patient reported Duchenne data set. In July, we had our highest number of new registrants since we launched on the App-based platform last year. Participating in the Registry is one of the easiest ways to help move research forward, and even learn about potential trials or therapies for you or your family members.

Supporting Newborn Screening

As we celebrate Duchenne Action Month, the global community also recognized Newborn Screening Awareness Month. After modifications to allow for remote recruitment in the face of COVID-19, PPMD’s Duchenne Newborn Screening Pilot continues to successfully enroll and screen babies as we approach the one-year mark since the first baby was screened late last year.

Developing Care & Support Resources

Since March, the PPMD Care Team has played an instrumental role in response to community concerns related to COVID-19, most recently about providing resources for back to school planning. We continue to add additional resources to PPMD’s COVID-19 Resource Center and host webinars and Facebook Live chats on topics the community is most concerned about.

We are growing our Certified Duchenne Care Center (CDCC) network, including the recent addition of Akron Children’s Hospital. This summer, each CDCC will receive a package of PPMD-branded care and support materials. These resources were created specifically for families dealing with a Duchenne diagnosis at each stage. We are also proud to report that the newly updated Education Matters is now available in three volumes according to age/educational level.

Maintaining a Robust Research Pipeline

Our Drug Development Pipeline remains robust. Even amid COVID-related delays, we have seen progress. Just last month we celebrated the news that the FDA granted accelerated approval to NS Pharma’s VILTEPSO™ (viltolarsen), a treatment of Duchenne in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This is the second FDA-approved targeted treatment for patients with this type of mutation and the fourth approval in the Duchenne community. We have updated our access resource center for families and will continue to work closely with regulators and payers as more Duchenne therapies become available.

I am so proud of the progress we continue to make. Despite the circumstances that have brought us to this place in time, we have found greater opportunities to connect with and support one another. I have truly seen the best in our community and continue to be impressed at how you, collectively, define resilience.

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