Three-dimensional microbrains built in the lab using stem cells from Rett syndrome patients can become a new model to study the disease, neuroscientists say. This can help identify new therapeutic compounds to treat Rett syndrome and accelerate therapy discovery for other neurodevelopmental disorders. The study, “3-Dimensional Human Cortical Neural…
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…
The first evidence that selective attention — the ability to focus on or select an object in the environment — is compromised by Rett syndrome was presented in new research findings. The article, “Impaired Visual Search in Children with Rett Syndrome,” was published in…
Meeting basic daily tasks, from getting dressed to assuring good hygiene and medical care, are so time-consuming for teenagers and young adults with Rett syndrome that time for more social and interactive activities is scarce, a study based on diaries kept by family members and caregivers found. Its researchers propose…
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…
Patient enrollment is now complete for the pivotal STARS study investigating the safety, tolerability, and effectiveness of sarizotan in Rett syndrome patients with respiratory abnormalities, Newron Pharmaceuticals announced. Top line results from the Phase 2/3 clinical trial are expected in the last quarter of 2019. Sarizotan was developed…
The Rett Syndrome Research Trust (RSRT) has awarded $10 million to a host of top international researchers to help find a cure for the neurological disorder. The 2018 award is the organization’s largest since its founding in 2008. To date, RSRT has awarded some $57 million for research. The…
A 6-month music therapy program improved social interaction and communication skills and reduced seizure occurrence among Rett syndrome patients, as well as relieving parental stress, a new study showed. The research article, “The effectiveness of music therapy for individuals with Rett syndrome and their families,” was published…
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…
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