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The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…
Dentists should be aware that children diagnosed with Rett syndrome can develop several oral symptoms that could affect their overall health and quality of life. The study with that finding, “Oral findings in Rett syndrome: An update and review of the…
Rett syndrome has a significant impact on the lives of those living and caring for children with this complex disorder, with caregivers likening it to an “obstacle course,” a study shows. Healthcare systems and health policies should take these findings into consideration to help improve the outcomes of those affected…
Like typically developing children, girls with Rett syndrome can distinguish known words from meaningless non-words when spoken to, but may do so using potentially peculiar neural processes, compared with their peers, according to a study. The research, “Spoken word processing in Rett syndrome: Evidence…
A high-fat diet can lead to extreme obesity in Rett syndrome patients due to excessive calorie intake, and to brain networks unable to fully control feeding behavior, a mouse study suggests. The easy availability of high-fat foods in modern diets may further compound the problem — whose root cause is genetic…
Researchers from the Maastricht University in the Netherlands have suggested a series of adaptations to help with the assessment of eye movements in patients with Rett syndrome. Their study, “Challenges in evaluating the oculomotor function in individuals with Rett syndrome using electronystagmography,” was published in the European…
Botulinum toxin treatment for excessive salivation — known as hypersalivation or sialorrhea — in Rett syndrome can effectively reduce saliva production and may also improve oral motor function, a study reports. The study, “The Treatment of Hypersalivation in Rett Syndrome with Botulinum…
After the U.S. Food and Drug Administration recently approved an investigational new drug application for Anavex 2-73, Anavex Life Sciences is proceeding with its Phase 2 clinical trial of the medication in Rett syndrome patients, the company announced. The trial (NCT03758924) will assess the safety, tolerability,…
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