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Clinical Research Alert: At-Home Research Study in People with DM1
Sanguine Biosciences, a provider of at-home clinical research services, is seeking people living with myotonic dystrophy type 1 (DM1) to participate in a natural history. The goal of this research is to enhance the understanding of DM1 to support development of new diagnostic and treatment options for people living with the condition. The study This study…
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MDA Ambassador Guest Blog: Built to Rise – Strength Forged Through Every Challenge
Darlene, who was diagnosed with spinal muscular atrophy (SMA) type 3 at age 19, just turned 55 years old. She has spent her life proving that challenges do not define her, but that perseverance does. Darlene has been blessed with 32 years of marriage, two amazing daughters, a wonderful son-in-law, and a thriving 20+ year career as a…
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How it feels to get recognition for my life as a caregiver
I’m no longer a young mom, but I was once. In fact, I was a very young first-time mom, as my oldest daughter, Lexi, 24, was born when I was 21 years old. Six more children followed: Max, 19; Chance, 18; Rowen, 16; Charlie, 14; Mary, 10; and Callie, 3. The four boys were all…
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Watch: Capricor Therapeutics – Regulatory Update & Clinical Insights on Deramiocel for Duchenne Cardiomyopathy
Capricor Therapeutics recently joined PPMD for a community webinar on Tuesday, July 29, 2025 to discuss the current status of Capricor’s Biologics License Application (BLA) for Deramiocel (CAP-1002). We discussed the regulatory implications of a Complete Response Letter (CRL), as well as provided information about cardiomyopathy in Duchenne, the HOPE-2 and HOPE-3 (Phase 3) clinical…
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Duchenne MD gene-editing therapy nets orphan drug designation
An experimental gene editing therapy for Duchenne muscular dystrophy (DMD) being developed by Precision Biosciences has received orphan drug status from the U.S. Food and Drug Administration (FDA). The designation focuses on treatments for rare diseases like DMD. Its benefits include tax credits and fee exemptions, along with seven years of market exclusivity if the…
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Community Voice: Why We Decided to Name MDA in Our Wills
Probably like you, recent years have altered our lives in ways both expected and completely unforeseen. Some of these changes have led us to review certain areas of our lives, especially our plans for the future. Donna Albrecht and her daughters, Katie and Abby (circa 1980). When our daughters were born, we saw a lawyer…
