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How I’ve gotten through a busy season in the life of my family
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 track of them, their well-being,…
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Marking ’30 Days of Strength’ for MD Awareness Month this year
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 established in 2019 by the Muscular Dystrophy…
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RAG-18 granted FDA orphan drug status for Duchenne, Becker MD
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 of dystrophin, the hallmark of both disorders. Orphan drug…
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Making memories on what was likely my last family vacation
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 Ernesto didn’t stir up waves…
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Elevidys, one year later: ‘It’s no longer just about coping with DMD’
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 patients after the U.S. Food and Drug…
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Living on campus is new territory for one son with DMD
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 provided for the first 18 years of their…
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Duchenne Added to Massachusetts Newborn Screening Panel
PPMD is excited to announce another significant milestone: Massachusetts has officially approved the addition of Duchenne to the state’s newborn screening panel! The addition of Duchenne to the Commonwealth’s newborn screen panel was included as an amendment to the Maternal Health bill (H. 4999) that was signed Friday, August 23, by Governor Healey and will…
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Champions in Dallas: An Evening of Culinary Delight and Philanthropy to Support CureDuchenne
Dallas’ Finest Will Unite to Advance Innovative Research and Care for Duchenne Muscular Dystrophy Dallas, TX – August 19, 2024– CureDuchenne, a leading nonprofit dedicated to finding a cure for Duchenne muscular dystrophy, is thrilled to announce its upcoming Champions in Dallas event. Set to take place on Thursday, October 3, 2024, at the stunning…
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FDA gives WVE-N531, exon 53 skipping therapy, supportive status
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 rare, defined in the U.S. as…
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For Duchenne survivors, dealing with grief provides us strength
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 first connected on social media over a poem…
