National Center for Advancing Translational Sciences

03/11/2026

As of March 11, 2026, this account is no longer active.

Please look for our content on the National Institutes of Health (NIH) page and follow along for the latest news and information from NIH!

02/13/2026

The National Institutes of Health (NIH) is hosting 2026 on Feb. 27, 2026. We invite patients, patient advocates, caregivers, health care providers, researchers, trainees, students, industry representatives and government staff to join us! Let’s unite in raising awareness & building connections in the community.

Register today and join us virtually or in person to learn more about progress in rare diseases research: https://go.nih.gov/C213uLm

02/02/2026

Because of a lapse in government funding, new posts and responses will not originate from this account until appropriations are enacted.

05/21/2025

A study supported by ’s Rare Diseases Clinical Research Network (RDCRN) collected data from people with amyotrophic lateral sclerosis (ALS) to better understand what biomarkers could help researchers track and predict the progression of .

Learn more about what they discovered, which could impact future trials and play a role in developing new treatments: https://go.nih.gov/3i2TfMA

05/13/2025

Amyotrophic lateral sclerosis (ALS) is a rare, progressive and incurable disorder that damages the brain and nervous system. A recent study supported by ’s Rare Diseases Clinical Research Network (RDCRN) showed how the level of a protein in the blood called neurofilament light (NfL) could help predict ALS survival and decline and might be used in designing new clinical trials to test potential treatments.

Learn more: https://go.nih.gov/3i2TfMA

05/08/2025

Colorectal cancer is the second-leading cause of cancer deaths in the United States. New research made possible through partial support from the Clinical and Translational Science Awards (CTSA) Program found that mailing stool-based tests with an offer for more personal follow-up with a patient navigator could increase screening in rural areas, where rates are typically low. These screenings have been shown to reduce the incidence of and mortality associated with colorectal cancer. Read more about this -supported research: https://go.nih.gov/hjQuE8S

04/30/2025

this research was supported in part by the ! Read more about the potential impact of this research. ⬇️

An artificial intelligence (AI) screening tool identified patients at risk for opioid use disorder and helped reduce readmission to hospitals.

03/27/2025

An NIH-supported study used an innovative approach known as a concise summary form to make the eConsent process more efficient. eConsent systems often make the clinical trial enrollment process clearer and faster than traditional in-person consent. The eConsent form could help future studies enroll participants in ways that are preferred by all.

Learn more about the study: https://go.nih.gov/e2wb4Zn

03/17/2025

Most are caused by a single gene mutation, making them potential candidates for gene-targeted therapies. These therapies can greatly improve health outcomes when the diseases are diagnosed early.

The -funded Newborn Screening by Whole Genome Sequencing ( ) Collaboratory is focused on enhancing early diagnosis of genetic conditions by screening newborns for rare diseases with available interventions. The initiative will explore the feasibility of integrating whole genome sequencing into United States public health newborn screening programs.
View the initiative page for details and learn more about its first : https://commonfund.nih.gov/venture/nbsxwgs

03/04/2025

A new study from Penn State Clinical and Translational Science Institute (CTSI) researchers found that an irregular schedule can have lasting effects on young people’s heart health, increasing risks later in life. These findings suggest that having a regular sleep cycle may be key to protecting adolescent .

This research was supported by NCATS’ , the National Institute of Mental Health and the National Heart, Lung, and Blood Institute (NHLBI).

Read more: https://www.psu.edu/news/research/story/timing-and-regularity-sleep-may-be-key-adolescent-heart-health

02/28/2025

are key to developing and evaluating new treatments for . We worked with the Eunice Kennedy Shriver National Institute of Child Health and Human Development to fund for Rare Diseases, Disorders and Syndromes grants to improve our understanding of rare diseases. These grants will also address other obstacles scientists face, like a lack of suitable biomarkers. Read more: https://go.nih.gov/yKGLcus

02/28/2025

Rare diseases have a home at NCATS, and we were proud to light up Building 1 at the center of the National Institutes of Health (NIH) campus in recognition of . NCATS Director Joni Rutter, Division of Rare Diseases Research Innovation Director Dominique Pichard, and the rest of our team stand in support of the community today and every day.