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Mashup Score: 0
Population-based estimates of survival among individuals with Duchenne muscular dystrophy (DMD) living in the United States are lacking. It is also un…
Source: www.sciencedirect.comCategories: Latest Headlines, PediatricsTweet
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Mashup Score: 7Study finds daily steroids safe and slow the progression of Duchenne muscular dystrophy - 2 year(s) ago
New research published in JAMA recommends daily steroid doses for children with Duchenne muscular dystrophy (DMD), marking a significant change in how the disease is treated. University of Rochester Medical Center (URMC) neurologist Robert Griggs, M.D., and Michela Guglieri, M.D., with Newcastle University in the U.K., led the study, which was conducted by a global team of researchers dedicated…
Source: medicalxpress.comCategories: General Medicine News, Latest HeadlinesTweet
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Mashup Score: 8Piezo1 possible key to supporting muscle regeneration in Duchenne Muscular Dystrophy - 2 year(s) ago
One protein, Piezo1, is key to marshalling muscle stem cells’ unique shapes and response to injuries, but it is in low supply in those with Duchenne muscular dystrophy, according to a team at the Perelman School of Medicine at the University of Pennsylvania. However, when they re-activated Piezo1, it allowed muscle stem cells in mice to return to their normal, distinctly-shaped states so that…
Source: medicalxpress.comCategories: General Medicine News, Latest HeadlinesTweet
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Mashup Score: 1Activation of the ubiquitin-proteasome system contributes to oculopharyngeal muscular dystrophy through muscle atrophy - 2 year(s) ago
Author summary Oculopharyngeal muscular dystrophy (OPMD) is a genetic disease characterized by progressive weakness of specific muscles, leading to swallowing difficulties (dysphagia), eyelid drooping (ptosis) and walking difficulties at later stages. No drug treatments are currently available. OPMD is due to mutations in a nuclear protein called poly(A) binding protein nuclear 1 (PABPN1) that is…
Source: journals.plos.orgCategories: Genetics, Latest HeadlinesTweet
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Mashup Score: 4
A clinical trial at UC Davis Health and six other sites showed that a cellular therapy offers promise for patients with late-stage Duchenne muscular dystrophy (DMD), a rare genetic disorder causing muscle loss and physical impairments in young people.
Source: medicalxpress.comCategories: General Medicine News, Latest HeadlinesTweet
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Mashup Score: 26New treatment could benefit up to 45 percent of patients with Duchenne muscular dystrophy - 2 year(s) ago
Up to 45% of patients with the most common inherited neuromuscular disease could benefit from a new “cocktail” drug being developed at the University of Alberta, according to research published this week in the Proceedings of the National Academy of Sciences.
Source: medicalxpress.comCategories: General Medicine News, Latest HeadlinesTweet
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Mashup Score: 7
Each year, about 20,000 children are diagnosed with Duchenne muscular dystrophy, a rare genetic condition that causes progressive muscle weakness and other systemic damage.
Source: medicalxpress.comCategories: General Medicine News, Latest HeadlinesTweet
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Mashup Score: 5
Researchers say study results point to the “high therapeutic potential” of targeting the sphingolipid synthesis pathway as a strategy against muscular dystrophy.
Categories: Future of Medicine, Latest HeadlinesTweet
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Mashup Score: 2
Sphingolipids accumulate in dystrophic muscle.
Source: Science AdvancesCategories: Healthcare Professionals, Latest HeadlinesTweet
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Mashup Score: 9Blocking sphingolipids counteracts muscular dystrophy - 2 year(s) ago
EPFL scientists have made the first link between muscular dystrophy and a group of bioactive fats, the sphingolipids, which are involved in numerous cell functions and other diseases.
Source: medicalxpress.comCategories: General Medicine News, Latest HeadlinesTweet
Using data from CDC’s MD STARnet, researchers found several medical treatments and therapies that may help improve survival for people with #Duchenne #MuscularDystrophy. Read the scientific summary: https://t.co/6nIHq66RJj #RareDisorders https://t.co/elv778cAnK