GeneTx and Ultragenyx Announce Positive Interim Phase 1/2 Data on Investigational GTX-102 Demonstrating Improvement in Patients with Angelman Syndrome

SARASOTA, Fla. and NOVATO, Calif., Oct. 26, 2020 (GLOBE NEWSWIRE) — GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced positive interim data from the Phase 1/2 study of GTX-102. Preliminary results from the first five patients treated […]

Plant compound reduces cognitive deficits in mouse model of Down syndrome

The plant compound apigenin improved the cognitive and memory deficits usually seen in a mouse model of Down syndrome, according to a study by researchers at the National Institutes of Health and other institutions. Apigenin is found in chamomile flowers, parsley, celery, peppermint and citrus fruits. The researchers fed the compound to pregnant mice carrying […]

Research sheds light on earliest stages of Angelman syndrome

New research from North Carolina State University provides insights into the earliest stages of Angelman syndrome. The work also demonstrates how human cerebral organoids can be used to shed light on genetic disorders that affect human development. Angelman syndrome is a genetic disorder associated with delayed development, intellectual disability, speech impairment and problems with movement. […]

Childhood syndrome linked to COVID-19 causes profound immune changes

Researchers have uncovered how the immune system is altered in a rare COVID-19 related illness in children referred to as paediatric inflammatory multisystem syndrome (PIMS-TS). PIMS-TS is a rare syndrome which has emerged in a small number of children during the COVID-19 pandemic. The condition causes severe inflammation in blood vessels and can lead to […]

Scientists find strong evidence that wasting syndrome is the same for all organisms

An interdisciplinary team of Dutch researchers has discovered that wasting syndrome, a severe byproduct of tuberculosis, is the same for all humans and animals studied. The discovery offers new opportunities to investigate the still insufficiently understood condition. The scientists also developed a promising method for diagnosing and possibly even predicting tuberculosis. Professor of Molecular cell […]

A bad diet may cause polycystic ovary syndrome

A bad diet may cause polycystic ovary syndrome: Scientists find obese women with condition have more harmful gut bacteria than healthy females Scientists studied 58 teenage girls with PCOS and found guts were unbalanced Girls had more ‘bad’ bacteria linked to high levels of testosterone – a PCOS cause Colorado scientists behind study advise PCOS […]