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Give before midnight TONIGHT to double your impact in developing PPMD’s DCRN
Happy New Year’s Eve! We’re almost there. At midnight we will ring in a new year, one that I hope will bring with it incredible experiences, happy memories, and lots of love. I am passionate about the impact of the Dystrophinopathy Clinical Research Network. In my role as Director of Data and Technology Strategy at…
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Double your impact in advancing the research evolution by the end of 2024
As the clock winds down on 2024 and we prepare to ring in the new year, I am awestruck by this community’s accomplishments and full of hope for the future. We have important work yet to do and together, we can revolutionize how we study, develop, and rely on multiple therapies to improve outcomes. This…
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How my sons’ DMD progression has changed my outlook
In the past 12 months, our oldest daughter got married, our oldest son moved across town into a college dorm, another son started his final year of high school, and, most significantly, I returned to full-time work outside the home. Life is drastically different for our family than it was a year ago. The changes…
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Together, let’s advance the research evolution
For my fellow adults living with Duchenne and Becker, today’s reality is vastly different from the one we were diagnosed into. We are living longer lives and feeling stronger while doing so. Many of us are graduating from college or grad school, becoming professionals and in some cases, starting families. Today, I am so excited…
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Even with hearing aids, I still have difficulty in group settings
Facioscapulohumeral muscular dystrophy (FSHD) primarily affects muscles in the face, scapular region, and upper arms, hence the name. Progression is different for everybody, but the condition can eventually affect most, if not all, muscles in the body. As an additional “bonus,” FSHD also can cause nerve deafness, a condition that tends to make words sound…
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Life is about time and timing when living with Duchenne
As a family living with Duchenne, time is everything to us. Decades of trials and research have paved the way for a new era, ushering in a reality with a growing landscape of both approved and investigational therapies. As a community, we have an opportunity to move the science not only further, but faster by…
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How I measure my quality of life as someone with LGMD
Since I began writing for Bionews, the parent company of Muscular Dystrophy News Today, I’ve connected with many people living with chronic illness outside of my “comfort community” — those with forms of muscular dystrophy (MD). While I live with limb-girdle MD, I have many friends who live with other types of MD. We all…
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How I’ve learned to embrace the highs and lows of caregiving
In the past few weeks, as I’ve sat down at my laptop to write my columns, I’ve often wondered if readers think I’m crazy. One week my columns are full of positivity and gratitude for the things I’ve learned, and the next week they’re full of my fears, frustrations, and anxieties. The more I think…
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Percheron Therapeutics ends clinical trial in Australia and Europe after data indicates no benefits after 6 months of treatment with avicursen (ATL1102)
Australian company Percheron Therapeutics released topline 6-month data from its Phase 2 study of avicursen in non-ambulatory individuals with Duchenne. The trial did not meet its primary endpoint, and there were no clear trends toward benefits on secondary outcomes. Therefore, Percheron is discontinuing the trial, and will share more analyses next year. READ MORE IN THE…
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Over 20 new MDA grants will fund research into muscle diseases
The Muscular Dystrophy Association (MDA) has awarded nearly two dozen new grants to fund research into various forms of muscular dystrophy, including Duchenne and limb-girdle, as well as related muscle diseases. Altogether, the funding totals more than $5 million across 21 grants, according to a press release from the nonprofit. “The Muscular Dystrophy Association is…
