Gene-Based Therapy Restores Cellular Development and Function in Brain Cells From People with Timothy Syndrome

 

NIH-supported study shows potential treatment pathway for neurodevelopmental disorder.

In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range of bodily systems, leading to severe cardiac, neurological, and psychiatric symptoms as well as physical differences such as webbed fingers and toes.Read full story

Short-Term Incentives for Exercise can Lead to Sustained Increases in Activity

NIH-supported study shows certain perks can spur long-term behavior change in adults with cardiovascular disease risks

Adults with heart disease risks who received daily reminders or incentives to become more active increased their daily steps by more than 1,500 after a year, and many were still sticking with their new habit six months later, according to a study supported by the National Institutes of Health that published in Circulation.Read full story

April is National Donate Life Month

In April, we take the time to celebrate those who have saved lives through the gift of organ, eye, and tissue donation and transplantation.

Nationally, more than 100,000 people are waiting for a lifesaving organ transplant and another person is added to the waiting list every eight minutes. Read full story

Scientists Discover Potential Treatment Approaches for Polycystic Kidney Disease

Innovative disease modeling and gene editing techniques begin to answer long-standing questions.

Researchers have shown that dangerous cysts, which form over time in polycystic kidney disease (PKD), can be prevented by a single normal copy of a defective gene. This means the potential exists that scientists could one day tailor a gene therapy to treat the disease.Read full story

Analysis of Social Media Language Using AI Models Predicts Depression Severity for White Americans, but not Black Americans

NIH-supported study also found Black people with depression used different language compared to white people to express their thoughts on Facebook.

March 26, 2024 – Researchers were able to predict depression severity for white people, but not for Black people using standard language-based computer models to analyze Facebook posts.Read full story

HHS Releases Large Scale Water Utility Affordability Survey Report as Part of the First-Ever Federal Water Assistance Program

 

March 21, 2024 – Today, the U.S. Department of Health and Human Services (HHS), through the Administration of Children and Families (ACF), released the Understanding Water Affordability Across Contexts, LIHWAP Water Utility Affordability Survey Report. This report – PDF provides an analysis of the largest survey documenting rates, arrears, disconnections, and fees in one dataset in the country.Read full story

Experimental Gene Therapy for Giant Axonal Neuropathy Shows Promise in NIH Clinical Trial

Treatment for rare childhood disease was well tolerated and slowed loss of motor function.

An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health (NIH).Read full story