30 Days of MD: Miroslava Zerova
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30 Days of MD: Miroslava Zerova
Givinostat Wins Rare Pediatric Disease Designation; Phase 3 DMD Trial Fully Enrolled
Italfarmaco has now completed recruitment — delayed in the spring due to the COVID-19 pandemic — of boys with Duchenne muscular dystrophy (DMD) for its ongoing Phase 3 trial of givinostat, according to a top company officer. In another milestone, Givinostat has “just received” rare pediatric disease…
Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US
It took one year for Dona Krystosek to get a diagnosis for her son, Levi, after he was born. The family received three misdiagnoses of fatal diseases until they found out Levi has Jansen’s metaphyseal chondrodysplasia — an extremely rare form of dwarfism.  “The hardest…
Vamorolone Continues to Improve Motor Function in DMD Boys, Phase 2 Trials Show
The experimental therapy vamorolone can improve motor function significantly in boys with Duchenne muscular dystrophy (DMD), without stunting their growth and with fewer side effects compared to standard corticosteroids, according to 18-month data from a Phase 2 trial and its extension study. Findings were reported in the study, “…
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