My wife, Wendy, and I recently enjoyed a beach vacation in North Carolina with our family. That included our three children, Nicole, Jill, and Ryan, as well as their partners and our three grandkids, Iva, Julia, and Theo. Hurricane Debby moved out the day we moved in, and Tropical Storm…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to RAG-18, being developed as a potential treatment for both Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). RAG-18 is a small activating RNA (saRNA) therapy from Ractigen Therapeutics that’s designed to counteract the shortage…
My past two columns highlighted significant life events: my daughter’s wedding and my return to the workforce. I’m keeping that theme going today as well, noting my oldest son’s move into his college dorm! Having children leave to experience lives of their own, outside the home you’ve…
A little over a year ago, neurologist Sarah Wright administered Elevidys (delandistrogene moxeparvovec-rokl) to then 5-year-old Hiram Secrist, making him the first Duchenne muscular dystrophy (DMD) patient to receive the gene therapy outside of a clinical trial. Elevidys became the first and only gene therapy available for DMD…
Earlier this month, I attended the debut of Stephanie Esther Fam’s play “Absence” at Singapore’s Gateway Theatre Black Box. Fam, a leading Singaporean poet and playwright in disability-led theater, is also a close friend of mine. Although I had known of her work for some time, we…
The U.S. Food and Drug Administration (FDA) has given WVE-N531, an investigational therapy for Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping, a rare pediatric drug designation. This status aims to incentivize companies developing treatments for serious or life-threatening conditions that primarily affect children and are considered…
Roughly four decades into living with limb-girdle muscular dystrophy, I’ve developed numerous ways to thrive. Like many others, I believe in the power of a positive attitude. I try my best to project a positive outlook when communicating with others, whether it’s face to face or by…
I have seven children, and three of them are living with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15, and Charlie, 13. If you’re a caregiver or a parent, I’m sure you’ll understand how tired I am. I won’t peruse my past columns, but I’m sure…
Avidity Biosciences‘ delpacibart zotadirsen, or del-zota for short — formerly called AOC 1044 — was found to safely increase levels of dystrophin production to 25% of normal in people with Duchenne muscular dystrophy (DMD), according to new data from the EXPLORE44 trial. In the Phase 1/2 clinical…
Kevin Schaefer, the associate director of community content at Bionews, the parent company of this website, recently shared a thought-provoking article from Psychology Today titled “How Chronic Illness and Masculinity Intersect.” It has me thinking about what kind of man I am and want to be. The…
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