A friend in the DMD world makes a difference in my self-care

In my recent columns, I’ve shared several challenges we’ve faced in the past months of my family’s journey with Duchenne muscular dystrophy (DMD). My accessible van will be in the shop for the foreseeable future. I’ve returned to work after over a decade at home as a…

The U.S. Food and Drug Administration has granted rare pediatric disease designation to NS-050/NCNP-03, an exon-skipping Duchenne muscular dystrophy (DMD) treatment from NS Pharma. Treatments granted this designation are aimed at serious or life-threatening diseases affecting children and fewer than 200,000 U.S. patients. “We are grateful for this…

Atamyo Therapeutics has partnered with the Dion Foundation for Children with Rare Diseases to expand into the U.S. a clinical trial of ATA-200, a potential gene therapy for limb-girdle muscular dystrophy (LGMD) type 2C/R5 — dubbed LGMD2C/R5. The Phase 1b study (NCT05973630), designed to evaluate the…

As most of America’s children go back to school, I thought it’d be timely to offer readers my perspective, as a retired teacher and wheelchair user, on the beginning of an academic year. I began teaching middle school music and band in the fall of 1995. I was 22 years…

While observing World Duchenne Awareness Day over the weekend, I reflected on the journey my family and I have taken, which has been shaped by Duchenne muscular dystrophy (DMD). This year the Muscular Dystrophy Association (Singapore), or MDAS, celebrated the day during its annual Go the…

I walked home from school today. I’m almost 45 years old, yet today, with my backpack on, my empty lunch container stuffed inside, and a sweater tied around my waist, I walked home from school. As I pondered whether I was overthinking that image, my daughter Mary, 9, who goes…

Up to a year of treatment with DYNE-251, Dyne Therapeutics’ investigational exon 51-skipping therapy, led to improvements in motor function for boys with Duchenne muscular dystrophy (DMD), according to a clinical trial update. Based on these positive data from the Phase 1/2 DELIVER clinical trial (NCT05524883), Dyne…

A noninvasive quantitative MRI, or qMRI, was found to detect early muscle abnormalities among people with limb-girdle muscular dystrophy type R1 (LGMDR1), according to a small study from Europe. Many of the qMRI findings correlated with clinical assessments of muscle function and patient-reported activities. “Our findings revealed alterations in…

My life has recently been a whirlwind of activity. Now that the dust is finally settling, I’ve taken advantage of the relative quiet to take inventory of my family. An inventory of my family? What does that even mean? I have a large family. Inventory is how I describe keeping…

The muscular dystrophy (MD) community is poised to mark National Muscular Dystrophy Awareness Month with events throughout September to call attention to MD and related neuromuscular disorders — and to raise critical funds for the more than 300,000 U.S. families thought to be affected. The annual observance was…