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I am watching a child lose his independence for the third time
My son Charlie has recently been unable to get into his power wheelchair on his own, and I know I am watching him lose the ability to ambulate, as I did with two of my other sons. Charlie, 15, like his brothers Max, 20, and Rowen, 17, lives with Duchenne muscular dystrophy (DMD). I also…
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Clinical Research Alert: Phase 3 Study of Pridopidine in Individuals with ALS
Researchers at participating clinical research sites are seeking individuals with amyotrophic lateral sclerosis (ALS) for a phase 3 study (PREVAiLS). This study will evaluate the safety and efficacy of the investigational drug pridopidine to treat ALS. Pridopidine, which is being developed by Prilenia Therapeutics and Ferrer, has shown neuroprotective effects in multiple preclinical studies, as…
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CureDuchenne’s annual conference to highlight research, family support
CureDuchenne will host its annual Futures National Conference, a four-day event focused on bringing education, connection, and hope to the Duchenne and Becker muscular dystrophy (MD) community. The event will be held on May 21-24 at the JW Marriott Orlando, Grande Lakes. It will highlight the latest research and therapies for Duchenne and Becker, and feature…
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The Why Behind MDA’s Campaign to Support Family Caregivers
Carlee Weber and Nicole Lucas This month, the Muscular Dystrophy Association (MDA) is launching an important advocacy campaign urging Congress to advance policies that improve the lives of family caregivers in the neuromuscular community. At a time when policymakers are actively weighing changes to home and community-based services and the cost of caregiving continues to…
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Catch up on PPMD Together: Philadelphia
This past weekend, PPMD kicked off our 2026 PPMD Together series in Philadelphia, Pennsylvania. Individuals with Duchenne and Becker, their families, clinicians, and industry partners gathered for a day and a half full of connection and community. Attendees were welcomed Friday evening with a casual adult reception to connect and reconnect with familiar faces, while…
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With my mobility severely limited, I’m in dire need of a wheelchair
One problem dealing with a progressive disease like facioscapulohumeral muscular dystrophy (FSHD) is that there’s no way to predict the pace of disease progression. I think this is true for the aging process as well. In my case, it may also apply to the joint deterioration I’ve experienced following a horrifying automobile accident years ago.…
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Rethinking Exercise in Duchenne Muscular Dystrophy: What New PT/OT Research Means for Families
Emerging guidance offers cautious optimism—here’s what parents and caregivers should know For years, families affected by Duchenne muscular dystrophy have heard a consistent message: “be cautious with exercise, don’t overdo it.” That guidance came from a place of protection. Duchenne causes muscles to break down over time, especially under strain. Without clear evidence, exercise carried unknown risks, including how much strain…
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Building routines that support Duchenne care
Shalom Lim, who lives in Eastern Singapore, was diagnosed with Duchenne muscular dystrophy at 4 months old in 1996. He shares how early diagnosis shaped a lifelong approach to daily care and routines. The post Building routines that support Duchenne care appeared first on Muscular Dystrophy News.
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Setting expectations for treatment adherence in Duchenne care
Edward Smith, MD, a neurologist, explains why setting clear expectations around treatment benefits and risks can help patients and families stay engaged with care plans over time. The post Setting expectations for treatment adherence in Duchenne care appeared first on Muscular Dystrophy News.
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Simply Stated: Introduction to CIAO1-Related Neuromuscular Disorder
CIAO1-related neuromuscular disorder is an inherited condition that was first reported on in late 2023. It is caused by variants in the CIAO1 gene and primarily affects muscle function, though many affected people also experience problems with the nervous system. Much about the symptoms and progression of this newly discovered condition remains to be defined.…
