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Today Roche announced plans for the SHIELD DMD study, a phase II open-label study to investigate the use of satralizumab in ambulatory and non-ambulatory individuals with Duchenne. Satralizumab is a monoclonal antibody that inhibits interleukin-6 and is an approved therapy for adults with neuromyelitis optica spectrum disorder. According to Roche, with the understanding of the…

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I feel relaxed — refreshed, even. We’ve had almost two weeks at home without any appointments or travel. The kids are complaining of boredom, but I’m letting boredom reign! It’s a beautiful but rare occurrence for my family of nine. My husband, Jason, and I have seven children: Lexi, 23, Max, 18, Chance, 17, Rowen,…

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  As the school year wraps up, we are all looking forward to a fun-filled summer. It can be overwhelming to plan outings and activities considering accessibility and necessary accommodations to keep everyone safe. Along with your ideas and feedback, PPMD has compiled a list of ideas for all ages and interests:   Spend time…

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Satellos Bioscience has established a clinical advisory board, with experts in drug development and genetic muscle disorders, to help propel SAT-3247, an oral therapy candidate for Duchenne muscular dystrophy (DMD). “The formation of this clinical advisory board marks a major development step for Satellos as we continue our evolution in becoming a clinical stage drug…

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After three years of treatment with Capricor Therapeutics’ experimental cell therapy CAP-1002, people with Duchenne muscular dystrophy (DMD) continue to show benefits in arm and heart function, new data from the HOPE-2 open-label extension (OLE) study shows. Topline data from the Phase 3 HOPE-3 trial (NCT05126758), which is testing the therapy in DMD patients, ages…

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Capricor Therapeutics has announced positive 3-year results from the ongoing HOPE-2 open-label extension (OLE) study of CAP-1002 for the treatment of Duchenne. CAP-1002 is the company’s novel cell therapy. The HOPE-2 is a double-blind, randomized, placebo-controlled trial in ambulatory or non-ambulatory patients with Duchenne. It is the first trial to use the Performance of Upper…

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Read the full news release HERE Capricor Therapeutics, which received early funding from CureDuchenne, has announced positive 3-year results from the HOPE-2 open-label extension study.  Individuals receiving CAP-1002 continue to show upper-limb and cardiac benefits after 3 years of treatment, compared to an external control group of similar DMD patients.  Results from the pivotal Phase…

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What’s it like to have three children with Duchenne muscular dystrophy (DMD)? That’s the question I’m asked more than any other, as a mom and caregiver to three sons with DMD: Max, 18, Rowen, 15, and Charlie, 13. Of course, that question is only asked by parents or grandparents of children with Duchenne. I assume…

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In a column I wrote last month, I talked about finally deciding to try some physical therapy, despite more than a little pessimism that it would be effective. Actually, I’ve been struggling with this decision for over a year. I finally followed through and completed a five-week program of in-home physical therapy last week. I…

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An engineered transfer RNA (tRNA) molecule developed by hC Bioscience could be a fix for the protein that’s lacking in Duchenne muscular dystrophy (DMD), supporting the company’s ongoing efforts to identify a lead molecule that may treat the disease. In a study with a mouse model of DMD, the engineered tRNA resulted in the production…

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