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Duchenne Muscular Dystrophy: Clinical Presentation and Key Differentiators
The post Duchenne Muscular Dystrophy: Clinical Presentation and Key Differentiators appeared first on Muscular Dystrophy News.
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The Cascade of Pathophysiological Dysfunction in Duchenne Muscular Dystrophy
The post The Cascade of Pathophysiological Dysfunction in Duchenne Muscular Dystrophy appeared first on Muscular Dystrophy News.
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The Gowers’ maneuver in Duchenne
Daniel Guillen, MD, explains why the Gowers’ maneuver is often considered a hallmark sign of DMD. Learn how this movement pattern reflects proximal muscle weakness, why it commonly appears in young boys with Duchenne, and what clinicians look for when evaluating early signs of the disease. Why is the Gowers’ maneuver considered a hallmark sign…
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Early clues that can point to Duchenne
Daniel Guillen, MD, discusses some of the early signs that may raise suspicion for DMD, including elevated CK levels, speech delays, and abnormal laboratory findings. Learn why these clues can help support earlier recognition and diagnosis. If a young boy presents with speech delay and high creatine kinase (CK), should we still suspect DMD? The…
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The major turning point in Duchenne progression
Daniel Guillen, MD, discusses one of the most significant milestones in DMD: the loss of ambulation. Learn why this transition marks a major inflection point in disease progression and how it relates to respiratory function, heart health, and long-term outcomes. What marks the major inflection point in the DMD disease trajectory? The post The major…
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Cardiac disease in Duchenne
Daniel Guillen, MD, explains how DMD affects the heart muscle and why cardiac disease often progresses quietly over time. Learn why heart complications may go unnoticed in boys with Duchenne and why ongoing cardiac monitoring is essential. Why is cardiac involvement often called the ‘silent threat’ in DMD? The post Cardiac disease in Duchenne appeared…
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Guest Voice: Celebrating World Collagen 6 Myopathy Day
I know what you’re thinking: another awareness day. One more health observance to briefly acknowledge for 24 hours, then mindlessly scroll past once it’s over. But for ultra-rare neuromuscular conditions, awareness is everything. Disease-specific awareness days actually matter. They create exposure, support, belonging, hope, and collaboration that can have a lasting impact. I was especially…
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Massachusetts Begins Newborn Screening for Duchenne
As of today, Massachusetts has officially begun screening all newborns for Duchenne muscular dystrophy. This milestone comes after the passage of the state’s Maternal Health bill (H. 4999), which was signed into law by Governor Maura Healey on August 23, 2024. The law included a critical amendment funding the program and mandating the addition of…
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Dyne hopes to bring new treatment for DM1 to US market in 2028
Dyne Therapeutics expects to report top-line data in early 2027 from its Phase 1/2 ACHIEVE trial testing zeleciment basivarsen (z-basivarsen), an experimental treatment for myotonic dystrophy type 1 (DM1). The company announced that enrollment is now complete for this key group of patients. If the results are positive, Dyne plans to apply for accelerated approval…
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Avidity’s trial for individuals with Duchenne amenable to skipping exon 45 is aiming to start in the first half of 2027
Read Avidity’s Community Letter: Avidity Community Letter_DMD45 Program Update_June 2026Download The post Avidity’s trial for individuals with Duchenne amenable to skipping exon 45 is aiming to start in the first half of 2027 appeared first on CureDuchenne.
