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This content is password protected. To view it please enter your password below: Password: The post Protected: Honoring Every Journey in Duchenne and Becker: Advancing Therapies, Access, and Care For All appeared first on Parent Project Muscular Dystrophy.

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Navigating the holidays creates some logistical problems for my family. It’s not because there are nine of us. We might be a big family, but if we can all fit in our average-sized, ranch-style home, then we can fit in most homes. Having three power wheelchair users in the family does make getting into many houses…

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Progress in neuromuscular research has always depended on collaboration — scientists, families, advocates, and organizations uniting to accelerate the path to treatments. This spirit is at the heart of the Muscular Dystrophy Association’s latest announcement: nearly $2 million in new collaborative research grants awarded with seven partner organizations to advance breakthroughs across ALS, congenital myopathies,…

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Upsher-Smith Laboratories is launching a new corticosteroid treatment for people with Duchenne muscular dystrophy (DMD) in the U.S. ages 5 and older. The therapy, branded Kymbee, is an oral tablet formulation of deflazacort, a corticosteroid available for DMD patients under brand names including Emflaza. Patients taking Kymbee will have access to Upsher-Smith’s Promise of Support…

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With the busy holiday travel season, many members of the neuromuscular community may be wondering about the latest policy developments in accessible air travel. This blog provides a round-up of recent activity on the issue and news you can use as you make your own travel plans this winter. Remember that passengers with disabilities have…

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Last month, I attended “The Sun Will Shine,” a jukebox musical staged at The Star Performing Arts Centre in Singapore. I was invited by the SingHealth Patient Advocacy Network (SPAN), a collective of patients and caregivers who offer feedback to improve healthcare services. As someone living with Duchenne muscular dystrophy (DMD), being part of SPAN…

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Capricor Therapeutics today announced positive topline results from its pivotal Phase 3 HOPE-3 clinical trial evaluating Deramiocel, the company’s investigational cell therapy for the treatment of Duchenne muscular dystrophy. According to Capricor, the study met both its primary skeletal muscle endpoint, Performance of Upper Limb (PUL v2.0) and the key secondary cardiac endpoint, left ventricular…

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Bio: Sonali Gupta is an essayist, journalist, and audio producer. She holds a master’s degree in journalism from New York University and previously worked as an audio producer in Mumbai, where she lived for over a decade. Her writing focuses on health, disability, and culture, with work appearing in The New York Times — including…

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A regimen of three immune-suppressing medications may improve the effectiveness of gene therapy for people with Duchenne muscular dystrophy (DMD), allowing patients to receive treatment more than once and making it accessible to people who are currently ineligible, a mouse study showed. DMD is caused by mutations in the gene that encodes dystrophin, a protein…

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Fin de l’essai clinique de l’ataluren pour la dystrophie musculaire Duchenne : dix ans d’espoir et une profonde incertitude La fin essai clinique ataluren dystrophie muscululaire Duchenne a été annoncée pour décembre 2025. Depuis presque dix ans, plusieurs familles québécoises, dont la nôtre, participent à un essai clinique concernant l’ataluren (Translarna), un médicament développé par…

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