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Affected − but not defined − by muscular dystrophy: ‘We’re all the same, even if we don’t look like it’
06 Apr 2025 10:32 GMT
… years ago about living with Duchenne muscular dystrophy, a rare genetic disorder … his annual visit at the Duchenne Muscular Dystrophy Center at UMass Memorial … Viltepso solution, used to help Duchenne muscular dystrophy patients strengthen their muscles, …
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Scots children with muscular dystrophy denied free new life-extending drug
06 Apr 2025 03:35 GMT
… . Givinostat slows the progress of Duchenne muscular dystrophy (DMD) and is being provided … living with rare conditions like Duchenne Muscular Dystrophy, have access to safe and … other eligible boys access to Duchenne.
“The cost involved is relatively …
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Caregiver Burden and Value Assessments in Duchenne Muscular Dystrophy
03 Apr 2025 17:17 GMT
As innovative treatments emerge for Duchenne muscular dystrophy (DMD), the complexities of caregiver …
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A mother’s battle: Advocating for life-saving treatments for children with Duchenne muscular dystrophy
02 Apr 2025 18:01 GMT
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Sarepta’s Duchenne Gene Therapy Trials Paused in Europe Amid Patient Death Investigation
03 Apr 2025 19:05 GMT
… (ENVISION, NCT05881408) evaluating Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy, delandistrogene moxeparvovec …
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Roche halts testing of Sarepta Duchenne gene therapy in Europe
03 Apr 2025 18:23 GMT
… 16-year-old patient with Duchenne muscular dystrophy.
The European Medicines Agency asked … for a limited population of Duchenne patients in 2023 and then …
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New Study Shows Duchenne's Effect on the Brain Might Be Reversed
02 Apr 2025 20:00 GMT
… has shown that uncovered that Duchenne muscular dystrophy (DMD), primarily recognized for its …
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Capricor Therapeutics’ CAP-1002: Regulatory Progress, Clinical Outcomes & Investment Outlook
06 Apr 2025 14:20 GMT
… active FDA review for Duchenne muscular dystrophy (DMD) cardiomyopathy. The … first cell therapy for Duchenne muscular dystrophy and specifically the first … improved survival with ventilation.
• Muscular Dystrophy Association – Vamorolone (Agamree) Approval …
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Family hopes to take boys with life-limiting condition to Disneyland Paris
06 Apr 2025 05:36 GMT
… and Riker, are living with Duchenne muscular dystrophy, a severe condition causing progressive … the most common type of muscular dystrophy diagnosed in children, and primarily …
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Edgewise Therapeutics (EWTX): Among Unstoppable Stocks That Could Double Your Money
05 Apr 2025 21:40 GMT
… studied for both Becker and Duchenne muscular dystrophy, and EDG-7500 for cardiomyopathy …