Feb 07, 2018
Children affected by prenatal drinking more numerous than previously estimated
Findings suggest rates of fetal alcohol spectrum disorders may be similar to autism spectrum disorders.
Jan 30, 2018
Researchers reverse symptoms in neurologic disease model
Study provides hope for Rett syndrome patients and others across autism spectrum.
Jan 04, 2018
X chromosome reactivation could treat Rett syndrome, other X-linked disorders
Mass. General-developed approach reactivates silenced chromosome in cell lines, animal model.
Oct 04, 2017
Study pokes holes in fetal alcohol hypothesis
Study challenges theory that cells in the brain's immune system are the culprit behind the neurologic damage that occurs in children exposed to alcohol while in the womb.
Jul 18, 2017
Reversing fetal alcohol damage after birth
Two commonly used drugs can repair damage caused by alcohol in utero
Apr 05, 2017
New step toward treatment of myotubular myopathy gene therapy
In a study published today in Molecular Therapy, researchers demonstrated the efficacy of administration of a therapeutic vector by a single intravenous injection and identified the dose that restores long-term muscular strength in a large animal model of the disease.
Dec 27, 2016
Experimental therapy for Prader-Willi syndrome shows promise in mice
Drugs capable of activating silenced genes, namely UNC0638 and UNC0642, were found to improve survival and growth outcomes in a mouse model of Prader-Willi syndrome according to a study funded by the National Institutes of Health.
Dec 20, 2016
Findings in tuberous sclerosis complex on the role of inflammation and epilepsy
A preclinical study provides a proof-of-concept suggesting that anti-inflammatory treatment could be a potential therapy for epilepsy in tuberous sclerosis patients. The study found rapamycin minimally corrected some brain tissue abnormalities in a mouse model, but, more importantly, there was a small decrease in seizure development and slightly improved survival in the mice.