-
Introducing PPMD’s Dystrophinopathy Clinical Research Network (DCRN): Advancing the Research Evolution
In our rapidly evolving therapeutic landscape, there is a critical need for cohesive infrastructure that helps track what happens when individuals are on more than one therapy and how we can continue to evolve standards of care in this new era. This is why PPMD is proud to introduce and begin development of the Dystrophinopathy…
-
Avidity Biosciences Announces Plans to File Biologics License Application for Delpacibart Zotadirsen (AOC 1044)
Avidity Biosciences, Inc. has announced its plans for filing a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for delpacibart zotadirsen (del-zota) for treatment of individuals living with Duchenne muscular dystrophy mutations amenable to exon 44 skipping. Del-zota (AOC 1044) is an exon skipping therapy conjugated to a monoclonal antibody targeting…
-
Gene-editing therapy for LGMD shows promise in mouse model
A gene-editing therapy designed to correct a defect in the DYSF gene — one that’s associated with a form of limb-girdle muscular dystrophy, or LGMD — was found to restore dysferlin protein levels and help regenerate muscle tissue in a mouse model of the disease. The researchers are now seeking funding to test the gene-editing approach…
-
PPMD’s Race to End Duchenne Program Celebrates 20 Years of Going the Distance to End Duchenne
PPMD is celebrating the 20th anniversary of our endurance fundraising program, Race to End Duchenne, this weekend in Orlando, Florida. 185 team members will participate in the 2025 Walt Disney World® Marathon Weekend, presented by State Farm, running in honor of loved ones affected by Duchenne. The Walt Disney World® Marathon Weekend is a special…
-
How ‘The Remarkable Life of Ibelin’ moved me as a DMD survivor
A few days ago, I watched the Netflix documentary “The Remarkable Life of Ibelin.” I wasn’t prepared for how deeply it would move me. The film follows Mats Steen, a young man from Norway who lived with Duchenne muscular dystrophy (DMD). Online, he was known by the avatar Ibelin, a hero in the game World…
-
Application finalized for DMD cell therapy CAP-1002, now deramiocel
Capricor Therapeutics has completed its submission of a biologics license application (BLA) seeking U.S. approval of deramiocel, the company’s cell therapy for heart muscle disease in people with Duchenne muscular dystrophy (DMD). “The submission of the BLA marks a pivotal step for Capricor and those impacted by DMD,” Linda Marbán, PhD, CEO of Capricor, said…
-
Thank You for Joining us to Advance the Research Evolution
As we enter PPMD’s fourth decade and a new era in Duchenne research, I am once again in awe of this community’s unwavering determination to push boundaries and drive progress. Your generosity continues to propel our initiatives forward in the fight to end Duchenne. More individuals living with Duchenne have access to therapies than ever…
-
Capricor completes BLA submission to the FDA for the approval of its investigational cell therapy for Duchenne cardiomyopathy
Capricor Therapeutics, which received early funding from CureDuchenne, has completed a Biologics License Application (BLA) with the US FDA, seeking full approval of deramiocel for individuals with Duchenne cardiomyopathy. Deramiocel (CAP-1002) consists of cardiosphere-derived cells which secrete extracellular vesicles known as exosomes, to promote immunomodulatory and antifibrotic actions. Link to press release: https://www.capricor.com/investors/news-events/press-releases/detail/303/capricor-therapeutics-completes-submission-of-biologics The post Capricor…
-
Capricor Therapeutics Completes Submission of Biologics License Application to the U.S. FDA for Deramiocel
PPMD is excited to learn that Capricor Therapeutics has completed the submission of its rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking full approval for deramiocel (CAP-1002) for the treatment of Duchenne muscular dystrophy cardiomyopathy. Deramiocel is Capricor’s novel cell therapy thought to decrease inflammation and fibrosis while exerting…
-
My husband and I make a great team as we care for our sons
Occasionally I struggle to find a topic to write about in my weekly column. I had that experience before writing this one, so I asked my husband, Jason, what I should write about. He jokingly said, “Husbands!” At first, I laughed with him, but I quickly realized it was a good idea. Jason and I…
