'No beds, not enough doctors': UK healthcare crisis laid bare
Five-year-old Yusuf Mahmud Nazir died from pneumonia after being sent home, despite a doctor describing his tonsillitis as the worst he had ever seen.
Jan 18, 2023
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Congenital, Hereditary, and Neonatal Diseases and Abnormalities: News and Research on Muscular Dystrophies
MeSH tree: C16.320.577
Using RNA interference to correct a mistake in the genetic code of patients with Fukuyama muscular dystrophy
Muscular dystrophy is a debilitating disease that causes progressive weakening and loss of muscles. Fukuyama congenital muscular dystrophy (FCMD), the second most common form of childhood muscular dystrophy in Japan, is a ...
Dec 12, 2022
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Death in US gene therapy study sparks search for answers
The lone volunteer in a unique study involving a gene-editing technique has died, and those behind the trial are now trying to figure out what killed him.
Nov 4, 2022
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Myotonic dystrophy: Mouse model shows that GABA receptors are implicated in sleepiness
People with the inherited disorder myotonic dystrophy (DM) often experience excessive daytime sleepiness and fatigue, as well as altered responses to anesthetics that can put them at risk for complications when hospitalized.
Sep 29, 2022
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Scientists studying fatal muscle wasting disease make significant discovery
Duchenne muscular dystrophy (DMD) is caused by a genetic mutation and affects one in every 5,000 boys born. Because the affected gene is on the X chromosome, girls are carriers of the mutant gene but develop the disease only ...
Sep 27, 2022
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Blocking an ion channel improves muscle function and survival in mice with severe Duchenne muscular dystrophy
Researchers at Johns Hopkins Medicine report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop a severe form of Duchenne ...
Sep 13, 2022
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Vamorolone effective, safe for boys with Duchenne muscular dystrophy
For boys with Duchenne muscular dystrophy (DMD), vamorolone, a structurally unique dissociative steroidal anti-inflammatory drug, is effective and safe over a 24-week period, according to a study published online Aug. 29 ...
Sep 1, 2022
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Not all in the genes: Are we inheriting more than we think?
A fundamental discovery about a driver of healthy development in embryos could rewrite our understanding of what can be inherited from our parents and how their life experiences may shape us.
Aug 11, 2022
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