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DMD carrier with heart failure has healthy pregnancy: Case study
A woman who developed heart failure due to Duchenne muscular dystrophy (DMD) was diagnosed and treated before she got pregnant, which led to a healthy outcome for her and her baby girl, according to researchers in Japan. The woman’s case was reported in “Successful Pregnancy Outcome With Preconception Care in a Symptomatic Carrier of Duchenne…
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Couch shopping awakened me to a focus on my children with DMD
I think about things deeply, rolling ideas around in my head until they make sense. My creative side, the writer, likes to wonder in this way. And as an introvert who naturally tends to keep everything to myself, writing is often the way I can express ideas and feelings after they’ve been processed. Reflecting on…
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This Year, I Am Grateful For Our Journey Together
As I prepare for another holiday to begin, I am again overwhelmed with gratitude for the compassionate, resilient community we’ve built through PPMD. You are each an extension of my family, the family we have created together. You bring light and hope for the future as we continue to fight. Over the past three decades,…
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Equitable Access in Clinical Trials—A Call for Action and Collaboration
Living with Duchenne and Becker presents unique challenges, especially for families from underserved or marginalized communities. Health disparities, delayed diagnoses, and limited access to care and clinical research create barriers that often result in diminished clinical outcomes. However, there is a growing sense of hope: by working together across various sectors, we can make meaningful…
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A walker should help prevent falls, right?
I’m getting more comfortable using my walker. However, it just doesn’t pay to trust the darned thing. The other day in my bedroom, I was putting clothes away. I was planning to take a shower later in the day, so I thought I’d save myself some steps. I grabbed a fresh set of clothes and…
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DMD gene therapy GNT0004 set to enter Phase 3 trial in Europe, US
GNT0004, an experimental gene therapy for Duchenne muscular dystrophy (DMD), appears to be working as intended in the initial parts of a multiphase clinical trial, with benefits including stable or improved motor function. That’s according to data presented by Genethon, the therapy’s developer, at the ASGCT Breakthroughs in Muscular Dystrophy conference, held in Chicago earlier…
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Taking control when living with limb-girdle muscular dystrophy
The power to influence or even direct people’s behavior or the course of events is part of a typical definition of “control.” When living with a chronic illness, as I am with limb-girdle muscular dystrophy, our desire to control the course of events can be a creative balancing act. We hope it’s like riding a…
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Changes this year helped me learn my limitations as a caregiver
Life feels busy, which is no surprise to me. My husband, Jason, and I share seven children: Lexi, 23; Max, 19; Chance, 17; Rowen, 15; Charlie, 13; Mary, 10; and Callie, 2. Max, Rowen, and Charlie live with Duchenne muscular dystrophy (DMD). Our lives are busy year-round. This year seems especially hectic because I returned…
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Regenxbio starts pivotal trial testing DMD gene therapy RGX-202
Regenxbio has initiated the pivotal phase of a clinical trial testing its experimental gene therapy RGX-202 in boys with Duchenne muscular dystrophy (DMD). If the results are positive, they could support the therapy’s accelerated approval by the U.S. Food and Drug Administration (FDA). Regenxbio has also announced new data showing RGX-202 treatment led to physical…
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How I experienced inclusion (and didn’t) during my education
On Saturday, I presented at the Participation — Inclusion in Action Conference 2024, which was held in my home of Singapore for the first time. My physiotherapist at the National University Hospital, who’s also a disability and healthcare researcher and one of the event’s key organizers, invited me to participate in February. In my talk,…
