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CureDuchenne was an early investor in Dyne Therapeutics in 2020, and congratulates the company for receiving Breakthrough Therapy Designation from the US FDA for DYNE-251, their exon 51-skipping agent for the treatment of Duchenne.  Why is this important:  The FDA grants Breakthrough Therapy Designations to speed up the development and review of drugs for serious conditions when preliminary clinical…

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Dyne Therapeutics has announced FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy. This designation reflects encouraging early results from the DELIVER clinical trial, where patients demonstrated sustained functional improvements, such as faster time to rise and improved walking speed over an 18-month period. DYNE-251 is designed to enable the production of near full-length…

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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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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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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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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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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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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…

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Spinal muscular atrophy (SMA) is a rare genetic disease affecting 1 in 11,000 live births in the United States. SMA is an autosomal recessive condition, meaning that a person with the condition receives two copies of the mutated SMN1 gene, one from each parent. The SMN1 gene is responsible for making SMN protein, which promotes…

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Last summer, I wrote a column about my beach vacation with my family. It was an incredible trip, but I had my doubts about future travel. With my facioscapulohumeral muscular dystrophy (FSHD) progression accelerating, I had to acknowledge that future travel might be difficult, if not impossible. My daughter Jill is our family vacation coordinator. She…

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