MeSH tree: C16.320.322
Rett syndrome is a rare neurodevelopmental disorder for which there is currently no cure or good therapy. It causes severe physical and cognitive symptoms, including many that overlap with autism spectrum disorder.
May 2, 2024
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A study in mice suggests how chromosome inactivation may protect girls from a type of autism disorder inherited from their father's X chromosome.
Apr 30, 2024
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Pharmaceutical giant Pfizer has received US approval for a gene therapy against a form of hemophilia, a rare and inherited blood clotting disorder, the company said Friday.
Apr 26, 2024
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Chronic kidney disease (CKD) is extremely prevalent among adults, affecting over 800 million individuals worldwide. Many of these patients eventually require therapy to supplement or replace kidney functions, such as dialysis ...
Apr 3, 2024
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The U.S. Food and Drug Administration has approved Duvyzat (givinostat) as an oral medication for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older.
Mar 26, 2024
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A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, or muscular dystrophies. This is shown in a new study carried out at Umeå University, Sweden, ...
Mar 6, 2024
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Fragile X syndrome is one of the most commonly inherited forms of autism and intellectual disability, and no treatment currently exists. But a team of University of Illinois researchers led by Vipendra Kumar, a postdoctoral ...
Mar 6, 2024
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Purdue University researchers have developed a patented therapeutic strategy for Lowe syndrome, an incurable and rare genetic disorder, by repurposing two drugs previously approved by the U.S. Food and Drug Administration ...
Feb 29, 2024
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How does coronavirus spread through the body? A new study, available on the preprint server bioRxiv, can help us answer that question. Professor Jakob Nilsson from the Novo Nordisk Center for Protein Research is one of the ...
Feb 27, 2024
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The drug vamorolone (Agamree) has been hailed as a promising new drug to treat Duchenne muscular dystrophy (DMD). It has recently been approved for clinical use in the United States by the Food and Drug Administration (FDA) ...
Jan 25, 2024
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