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I’ve learned to embrace assistive equipment for my sons with DMD
I spent part of this week with three of my sons in Denver. On Wednesday, they had a clinical trial appointment, followed by an appointment at the assistive technology clinic to try out some robotic arms. In anticipation of the latter, I began to reflect on my sons’ journey with medical equipment. I have three…
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Clinical Study Alert: Phase 2 Study of Brogidirsen in Boys with DMD
Researchers at NS Pharma are seeking boys with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping therapy to participate in a phase 2 clinical study (clinicaltrials.gov ID: NCT05996003) of their investigational therapy brogidirsen (NS-089/NCNP-02-201). DMD is caused by gene mutations that lead to the loss of the dystrophin protein, which is important for the structure…
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Scientists develop way to make DMD treatments work better
Researchers have developed a way to enhance the efficacy of exon-skipping therapies for Duchenne muscular dystrophy (DMD), and the approach showed promise in a mouse model of the disease. The study, “A Combinatorial Oligonucleotide Therapy to Improve Dystrophin Restoration and Dystrophin-Deficient Muscle Health,” was published in Molecular Therapy Nucleic Acids. DMD is caused by mutations in the…
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Living with Duchenne muscular dystrophy but choosing opportunity
In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, X, and Pinterest for more stories like this, using the hashtag #MDSpotlight,…
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MDA Advocacy Team Reports Updates in Newborn Screening Legislation
This year has been an eventful year for newborn screening in neuromuscular disease, with potential further progress on the near horizon. We began 2025 with all 50 states screening for spinal muscular atrophy (SMA), meaning nearly all babies born in the United States can be screened for SMA leading to a rapid diagnosis and near…
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Understanding BiPAP Webinar: Why It’s Needed, What Gets in the Way, and, How to Increase Compliance
The PPMD team was recently joined by pulmonologist Dr. Janaki Paskaradevan, psychologist Dr. Allison Clarke, and Maria Gonzalez, MSN, APRN, FNP-BC, CPN, from the Division of Pulmonary and Sleep Medicine at Ann & Robert H. Lurie Children’s Hospital, where they led a discussion on the critical role of BiPAP use in Duchenne and Becker muscular…
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How I advocate for my community as a girl with Duchenne MD
In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, X, and Pinterest for more stories like this, using the hashtag #MDSpotlight,…
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Head Back-to-School in Style with Adaptive Fashion
For many families with kids, the end of summer includes preparations for heading back to school for a new year of growth, learning, and activities. On top of school supplies, schedules, and meeting new teachers and classmates – this time of year also usually means back-to-school clothes shopping. As the fashion industry continues to make…
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Exondys 51 may help protect DMD heart function: Study
Duchenne muscular dystrophy (DMD) treatment Exondys 51 (eteplirsen) may help protect the heart as well as muscles used for movement, a study found. The treatment slowed heart function decline, raising the possibility it could lower the risk of cardiomyopathy, a serious condition that can lead to heart failure. “This study is the first to demonstrate…
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Living with Duchenne has given me more freedom
In recognition of Duchenne Muscular Dystrophy Awareness Month in September, the Duchenne Muscular Dystrophy Community Spotlight campaign features a series of stories highlighting the real-life experiences of people affected by Duchenne muscular dystrophy, written in their own words. Follow us on Facebook, Instagram, X, and Pinterest for more stories like this, using the hashtag #MDSpotlight,…
