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DYNE-101 is leading to functional gains for DM1 patients in trial
The investigational therapy DYNE-101 worked as expected and led to functional improvements for people with myotonic dystrophy type 1 (DM1) in a clinical trial, according to an update from developer Dyne Therapeutics. The Phase 1/2 ACHIEVE trial (NCT05481879) will now enroll a new group of participants who will all receive the dose that’s so far…
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PPMD Advocacy in the 119th Congress and the 47th Administration
As the 119th Congress and the 47th Administration prepare to take office, PPMD is committed to furthering the needs of our community through comprehensive, creative, and tenacious advocacy on Capitol Hill, regardless of politics. Over the years, we have earned the trust and respect of legislators across the political spectrum to support our mission to…
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PPMD Hosts 2025 Duchenne Healthcare Professionals Summit in San Diego, California
PPMD is excited to host the sixth in-person Duchenne Healthcare Professionals Summit in San Diego, California, January 15-17. Since the Summit’s inception in 2018, the event has brought together thousands of healthcare providers, researchers, leaders in federal advocacy, and industry partners for discussion, collaboration, and connection. Over three full days of meetings, over 350 attendees…
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GIVI-MPC named FDA orphan drug for treatment of Becker MD
The U.S. Food and Drug Administration (FDA) has granted IPS Heart’s GIVI-MPC — a stem cell therapy designed to make new muscle tissue in people with muscular dystrophy, or MD — orphan drug status for the treatment of Becker muscular dystrophy (BMD). This designation aims to encourage the development of treatments for rare diseases, or those…
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How my FSHD progression leads to out-of-body experiences
Many times over the years since the 1989 car crash that nearly killed me, I’ve been asked if I had any near-death or out-of-body experiences while I hung between life and death. I always try to clarify what people mean by these terms. If we’re talking about my spirit hovering in the corner of a…
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My original diagnosis was incorrect. Here’s why that’s important.
In August 1985, I was diagnosed with Becker muscular dystrophy. At that time, there was no genetic test for the disease, nor DNA sequencing of the blood. Instead, I underwent a muscle biopsy on my left thigh and calf, the tissue was sent for pathology, and the medical professionals told my parents that while I…
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Dyne plans to submit for FDA Accelerated Approval for Duchenne exon 51 skipping in 2026; Trial still recruiting participants.
Dyne Therapeutics, which received early funding from CureDuchenne, plans to pursue US Accelerated Approval from the FDA in 2026 for DYNE-251, an exon-skipping experimental therapeutic for individuals with Duchenne amenable to skipping Exon 51. Data from their ongoing DELIVER trial, expected Q4 2025, will support this submission, and Dyne is currently enrolling participants for an…
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Waking up on Christmas Eve made me scared and anxious
The Christmas decorations are down and tucked into storage for another year. The house is clean, the counters are clear of the holiday sweets, and our routine is again in sync. However, this holiday season has left lingering fears and sadness quivering in my heart. I share seven children with my husband, Jason: Lexi, 23,…
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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…
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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…
