Our Goal
The goal of long-term follow-up (LTFU) is to assure the best possible outcome for individuals with disorders identified through newborn screening (NBS). The LTFU-Cares and LTFU-Check Initiative is a two-year effort to develop, implement, and evaluate LTFU for newborns diagnosed with Spinal Muscular Atrophy (SMA) through NBS.
Our Team
Amy Brower, PhD, Director, Principal Investigator
Dr. Brower has a background in medical genetics, genomics, informatics, FDA submissions, newborn screening, translational research, molecular diagnostics, and bioinformatics. Dr. Brower directs a team that develops resources and tools for the newborn screening community to collect, analyze, visualize, and share clinical and genomic research data to better understand genetic disease across the lifespan.
Chris Pitro, Chief Financial Officer (CFO)
Mr. Pitro will provide project guidance related to budget planning.
Melanie Wells, Chief Executive Officer
Ms. Wells will ensure the required operational and administrative functions are available as needed to accomplish the LTFU-Cares and LTFU-Check deliverables. She will also provide cross-coordination with related ACMG activities
Our Collaborators
Emma Ciafaloni, MD, FAAN
Dr. Ciafaloni will advise the formation of an expert workgroup of clinicians to develop the LTFU-Cares Dashboard. Dr. Ciafaloni will also help to prioritize surrogate measures of success that are important to clinicians and help to define care coordination through a medical home for SMA.
Bo Hoon Lee
Department of Neurology, University of Rochester, NY
Jennifer M. Kwon
Department of Neurology, University of Wisconsin School of Medicine and Public Health, Madison, WI
Megan Waldrop
Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children’s Hospital, Columbus, OH and; Departments of Neurology and Pediatrics, Ohio State University Wexner Medical Center, Columbus, OH
Russell J Butterfield
Department of Pediatrics and Neurology, University of Utah, Salt Lake City, UT
Geetanjali Rathore
Division of Neurology, Department of Pediatrics, University of Nebraska College of Medicine, Omaha, NE
Aravindhan Veerapandiyan
Division of Neurology, Department of Pediatrics, University of Arkansas for Medical Sciences,
Arkansas Children’s Hospital, Little Rock, AR
Kapil Arya
Division of Neurology, Department of Pediatrics, University of Arkansas for Medical Sciences,
Arkansas Children’s Hospital, Little Rock, AR
Julie Parsons
Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO 80045
Melissa Gibbons
Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO 80045
Our Funder
The Health Resources and Services Administration (HRSA) Maternal & Child Health (MCH) Bureau works to improve the health and well-being of America’s mothers, children, and families. In partnership with states and communities, the Bureau supports health care and public health services for an estimated 55 million people nationwide. MCH funds several programs and efforts related to newborn screening (NBS). The LTFU-Cares and LTFU-Check Initiative is one of six efforts funded as part of the MCH Long-term Follow-up for Severe Combined Immunodeficiency and Other NBS Conditions program.
The goal of this program is to ensure that newborns and children identified through NBS achieve the best possible outcomes by expanding the ability of state public health agencies to provide screening, counseling and services to these newborns and children and to collaborate with clinicians, public health agencies and families to create a system of care that can assess and coordinate follow-up and treatment of newborn screening conditions. (link to the funding opportunity: HRSA-21-079)
PURPOSE
- To support the development of comprehensive models of LTFU that demonstrate collaborations between clinicians, public health agencies, and families;
- To ensure that newborns and children identified through NBS achieve the best possible outcomes.
GOALS
- Expanding the ability of state public health agencies to provide screening, counseling, and services;
- Collaborating with clinicians, public health agencies and families to create a system of care that can assess and coordinate follow-up and treatment of NBS conditions.
OBJECTIVES
- Implement LTFU model protocols with at least five clinical sites;
- Demonstrate integration of LTFU model data from clinical and public health systems;
- Increase the number of infants, children, and families who receive coordinated LTFU care through a medical home.
OUTCOME
- LTFU models supported under this HRSA program should facilitate the collection and integration of data from public health and clinical information systems to assess, inform, and ultimately achieve comprehensive LTFU of individuals identified through NBS.