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Making adaptations while living with Duchenne muscular dystrophy
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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An infected bug bite landed me in the ER, but I was in great hands
My daughter Jill and my wife, Wendy, are my primary caregivers. In August, Jill and Wendy traveled to Iceland together. The day before their departure, we had a “bon voyage” family picnic. It was a lovely gathering, but I ended up with a couple of bug bites on my left calf that evening. Regrettably, those…
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Quest Podcast: Precision Medicine: Mapping the Genetic Code for New Treatments
In this Quest Podcast episode, we chat with Dr. Stephan Züchner, Dr. Conrad “Chris” Weihl, and the Interim Chief Research Officer of the Muscular Dystrophy Association, Dr. Angela Lek. Leaders in the field of genetic mapping, all three have devoted their time and expertise to research and treatments for neuromuscular diseases. Their goal is to…
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MDA Summer Camp Alumni Put the Power in Powerhockey
Philadelphia Flyers PowerPlay Orange Team takes the court in Game 3 of the tournament. Photo credit: Bradley Digital. On the weekend of August 1, five Powerhockey teams from across North America hit the arena to compete for victory in the 2025 Powerhockey Cup. Hosted by the Philadelphia Flyers PowerPlay at Neumann University in Aston, PA,…
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Insights by Ira: From Telethons to Treatments – Decades of Impact
When I think of Labor Day Weekend every September, it truly invokes so many emotions and memories for me! As a 90’s kid from the mid-west, the holiday meant it was the official start to another school year. It meant that summer was over, and fall was upon us. But growing up, for me and…
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Exon-skipping DMD therapy gets FDA orphan drug status
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to NS-051/NCNP-04, an exon-skipping therapy designed to treat certain people with Duchenne muscular dystrophy (DMD). The FDA gives this designation to experimental treatments designed to improve care for rare disorders, specifically conditions affecting fewer than 200,000 people in the U.S. The goal is…
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Being part of a disabled community means learning to trust
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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An open letter to doctors who aren’t familiar with limb-girdle MD
Dear doctors, I am writing to share my knowledge about living with a rare, chronic condition called limb-girdle muscular dystrophy. There are many types of muscular dystrophy (MD), ranging in age of onset, severity, and common symptoms and comorbidities, such as heart and lung involvement. But regardless of the type, MD’s social, emotional, and mental…
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Ready for Respiratory Season: Keeping You and Your Family Healthy
With the start of school and the approach of winter, respiratory viruses are expected to rise. This year, and every year, it’s important to protect yourself, your children, and your family from respiratory infections. This is especially true for more serious illnesses like influenza (flu), RSV, and COVID-19. For individuals with Duchenne or Becker muscular…
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WEBINAR: Avidity EXPLORE44-OLE Functional Data Update
Avidity reports multiple functional improvements with Del-zota for individuals with Duchenne amenable to skipping exon 44. Watch HERE The post WEBINAR: Avidity EXPLORE44-OLE Functional Data Update appeared first on CureDuchenne.
