Huntington’s disease (HD), a devastating inherited neurodegenerative disorder, typically manifests in adulthood, progressively impairing motor functions, cognitive capabilities, and overall brain function over approximately 15 to 20 years post-diagnosis. Despite the discovery over three decades ago
In a significant step toward understanding and combating childhood cancers and congenital disorders, the Gabriella Miller Kids First Pediatric Research Program (Kids First), an initiative of the National Institutes of Health (NIH), has released two comprehensive new datasets. Focusing on childhood
Sangamo Therapeutics, Inc., a leading genomic medicine company, has reported promising updated data from the Phase 1/2 STAAR study. This study explores the efficacy of isaralgagene civaparvovec (ST-920) as a treatment for Fabry disease. The analysis, which reflects data up to September 12, 2024,
In a surprising move, Rentschler Biopharma announced its decision to withdraw from the cell and gene therapy (CGT) business at its Stevenage, UK facility, marking a significant shift in the company's strategic focus. This decision comes despite recent investments in building advanced capabilities
A recent study conducted by researchers at NYU Langone Health has reexamined the purported link between a mother’s health during pregnancy and the likelihood of her child developing autism. This study disputes previous assertions that maternal health conditions during pregnancy contribute directly
The groundbreaking research conducted by Van Andel Institute scientists sheds new light on how developmental epigenetic changes can profoundly influence cancer risk later in life. The study, published in Nature Cancer, identifies two distinct epigenetic states formed during development, which are
