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MDA Ambassador Guest Blog: Strong Enough to Carry Grief AND Gratitude with FSHD
Skye Anderson lives in North Carolina, and is an advocate living with facioscapulohumeral muscular dystrophy (FSHD). She is passionate about accessibility, spreading awareness, and empowering others with disabilities to use their voice. In Skye’s free time, she enjoys expressing herself through art, cheering on the Carolina Hurricanes, and connecting with others through shared experiences. Adaptive…
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cTAP Announces Novel Prognostic Score Developed for Duchenne Muscular Dystrophy Patients Offers Improved Prediction of Loss of Ambulation
As a founding partner and initial funder of the Collaborative Trajectory Analysis Project (cTAP), CureDuchenne is proud to support an important advancement for the Duchenne community: a novel prognostic score that helps predict when an individual may lose the ability to walk. This advancement can help clinicians better anticipate care needs and support families while enabling more…
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MDA’s Guide to the Americans with Disabilities Act (ADA)
More than three decades after it became law in 1990, the Americans with Disabilities Act (ADA) remains one of the most important civil rights protections for people with disabilities in the United States. Designed to prohibit discrimination and expand access in everyday life, from workplaces and schools to transportation systems and public spaces, the ADA…
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FDA grants orphan drug, rare pediatric disease status to GEn-1123 for DMD
The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to GEn1E Lifesciences‘ GEn-1123 as a potential treatment for Duchenne muscular dystrophy (DMD). The designations aim to incentivize the development of treatments for rare disorders, which are those affecting fewer than 200,000 people in the U.S. Orphan drug status provides…
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CureDuchenne Invests in SonoThera to Advance Nonviral Gene Therapy Platform for Duchenne Muscular Dystrophy
SonoThera press release CureDuchenne is pleased to announce that it has invested in SonoThera, supporting the advancement of its targeted, ultrasound-mediated gene delivery technology focused on the delivery of full-length dystrophin. SonoThera’s approach is designed to address multiple key limitations of conventional AAV-delivered gene therapies such as restricted payload capacity (limited to the delivery of…
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Adding new adaptations around the home to allow my caregivers a break
If you read my last column, you’ll know that I’ve been experiencing extreme physical challenges in the last couple months. These changes are a result of a combination of my facioscapulohumeral muscular dystrophy, damage from a major automobile accident many years ago, and simply growing old. This has resulted in a lot more pressure on…
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Stable heart health seen long-term for women with BMD, DMD mutations
Among women who carry mutations that can cause Becker muscular dystrophy (BMD) or Duchenne muscular dystrophy (DMD), measures of heart health — important given that heart muscle damage is a key driver of death in most people with these muscular dystrophy (MD) types — are generally stable over time. Those are the findings of a…
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In Case You Missed It…
Quest Media is an innovative, adaptive lifestyle platform from MDA. With the power of this platform, we foster awareness and empowerment and have important conversations with experts, thought leaders, and members of the neuromuscular disease community about topics that matter to them and to the larger community of individuals with disabilities. With so many valuable…
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What stress management skills can we use in our daily lives with MD?
I recently read a New York Times article about stress. Whether you live with a disability or not, stress is a universal phenomenon. Life is full of stressful moments both large and small that can overwhelm the best of us. In the article, the author asks three questions designed to help readers process stress: Is…
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Current Treatments for Duchenne Muscular Dystrophy
The post Current Treatments for Duchenne Muscular Dystrophy appeared first on Muscular Dystrophy News.
