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Life with Lily #2: Advocating for Disability Rights
Advocacy doesn’t always look bold or dramatic. Most of the time, it’s quiet, messy, and uncomfortable. Long before the first time I ever walked into a Congressional office to lobby my representatives for legislation that would support Americans with disabilities, I was already advocating. I just didn’t realize it. What hadn’t occurred to me was…
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Analyses show benefits of DMD gene therapy Elevidys
The one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) was associated with stabilizations in motor function for boys with Duchenne muscular dystrophy (DMD) who were treated when they were 8 or 9 years old, according to new trial analyses. Boys with DMD at this age are typically expected to show functional declines, and these data from the…
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Understanding the Threats to the ACL – and What It Means for You
In late March, the U.S. Department of Health and Human Services (HHS) announced a ‘dramatic restructuring’ of the Department, including reorganization of the Administration for Community Living (ACL), an agency that supports people with disabilities and their right to live independently in the community. In the press release of the announcement, it was indicated that…
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How to turn weakness into strength in life with muscular dystrophy
In September 1995, I began my first teaching job at a New Hampshire public middle school instructing students about band instruments and music. At the time, I was 10 years into my diagnosis with Becker muscular dystrophy. I wouldn’t be rediagnosed — this time, correctly — with limb-girdle muscular dystrophy type 2E/R4 until 2012. I…
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How to Plan the Perfect Summer Staycation
As the cost of living continues to rise in the United States, many individuals and families are re-evaluating their budgets. Typically, vacations are among the first things to get cut when creating a plan to save money. But trying to stay on budget this summer doesn’t mean that you have to cut out all of…
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When things go wrong, try to maintain an attitude of gratitude
If you’ve read any of my recent columns, you know that I’ve had a lot of health struggles of late. I’m back on the medical merry-go-round with physical therapy, occupational therapy, medical appointments, and tests that tie me up multiple days every week. I’m struggling to stand up from a seated position and my right…
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DMD gene therapy Elevidys wins conditional approval in Japan
The Japanese Ministry of Health, Labour, and Welfare (MHLW) has conditionally approved the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) to treat some cases of Duchenne muscular dystrophy (DMD). The approval covers its use in children, ages 3-7, who don’t have deletions in exon 8 and/or exon 9 in the DMD gene and are negative for antibodies…
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Duchenne muscular dystrophy gene therapy
Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.
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Entrada receives authorization to start exon 44 skipping trial in Europe
CureDuchenne provided early funding to Entrada Therapeutics. Entrada has received regulatory authorization in Europe to initiate a Phase 1/2 clinical trial of ENTR-601-44 in ambulatory individuals with Duchenne amenable to skipping exon 44. The study, which will run in the UK and the EU, will start in the 2nd quarter of 2025. Entrada is also…
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MDA Ambassador Guest Blog: A Life of Service
Greg Dodson lives in Tennessee with his wife, Mona. He is a US Army Veteran who has logged 3,862 jumps from airplanes! He received a diagnosis of ALS in 2019, though his symptoms were apparent much earlier. He is very involved in his local community and loves to give back. Greg’s photo featured on the…
