The CTSA Program is designed to strengthen and support the entire spectrum of translational research from scientific discovery to improved patient care. Read the latest in CTSA Program news about underserved populations in translational research across the lifespan.
CTSA Program Investigators Collaborate to Enhance Newborn Screening
Efforts are now underway to add rare diseases to newborn screenings through Early Check, a research program in which voluntary screening for fragile X syndrome and other genetic conditions yet to be determined will be offered to up to 120,000 families each year in North Carolina. NCATS is supporting Early Check through a CTSA Program Collaborative Innovation Award (CCIA).
Unprecedented Trans-NCATS Collaboration Enables Rapid Advancement of Rare Lung Disease Therapy to Human Trials
NCATS helped shepherd a potential new drug for a deadly lung disease, called autoimmune pulmonary alveolar proteinosis, through many steps of the translational science process. Center support provided a bridge in moving the promising therapeutic from pre-clinical to clinical research.
CTSA Program-Supported Research Uncovers Genetic Components of Healthy Aging
Older adults often face aging-related ailments that can be costly and shorten lifespans. However, some people live long lives without encountering these common health problems. What sets apart healthy agers from their peers? Researchers at Scripps Translational Science Institute are conducting a study of the “Wellderly” to find out.
CTSA Program Supports Research Network in Geographically Isolated Region
With support from NCATS’ CTSA Program, the University of New Mexico Clinical and Translational Science Center was a founding partner in the Mountain West Research Consortium (MWRC), currently a network of 11 universities spanning seven states. The MWRC’s goal is to build and enhance the geographically isolated region’s clinical and translational research capacity.
CTSA Program Domain Task Forces: Spotlight on Integration Across the Lifespan
The CTSA Program Integration Across the Lifespan Domain Task Force goals are to integrate translational science across the entire lifespan to achieve health improvements, launch efforts to study special population differences in the progress and treatment of disease processes, and develop a seamless, integrated approach to translational science across all phases of research.
CTSA Program Resources Support Largest U.S. Newborn Screening Study for Fragile X Mutations
A team of researchers at the University of California, Davis, found that more people have gene changes linked to Fragile X syndrome than anticipated. Accurate and timely diagnosis by such newborn screening may enable early intervention.
Patients with Rare Muscle Disorder Benefit from Repurposed Heart Drug
Patients with nondystrophic myotonia, a rare genetic muscle disorder, often have problems with debilitating muscle stiffness, fatigue, and sometimes even episodes of paralysis. Now, these patients have new help: a drug called mexiletine, normally used to treat heart disorders. With assistance from NIH’s Rare Diseases Clinical Research Network and the CTSA Program, researchers at seven institutions in four countries collaborated to demonstrate in a clinical trial that the heart drug is effective in treating patients with this rare disease.
New Drug for Rare Type of Cystic Fibrosis
Patients with a rare type of the deadly lung disorder cystic fibrosis may be able to breathe easier thanks to a new drug that targets the defective protein causing the disease. Researchers from 13 universities and hospitals, including 10 CTSA Program institutions, partnered with the Cystic Fibrosis Foundation and the drug manufacturer Vertex Pharmaceuticals to conduct clinical trials and obtain Food and Drug Administration approval for the drug Kalydeco as a new treatment.