Transforming health care and outcomes for children with rare diseases is difficult within the current health care system. There is great variation in care delivery, inadequate and slow application of existing evidence, and ineffective use of available data to generate new knowledge. Individual care centers have inadequate numbers of patients for robust learning and improvement. In order to redesign the system, changes must take place at multiple levels, including the patient and family, clinician, practice and the network. The purpose of this project is to design, develop, and test further refinements to an improvement and research network focused on HLHS, the most severe congenital heart defect, and to use a registry to simultaneously improve clinical care, redesign care delivery systems and to conduct quality improvement, health services, outcomes, and comparative effectiveness research. The purpose of this initiative, specifically, is to improve care and outcomes for infants with HLHS by: 1) expanding the established NPC-QIC national registry to gather clinical care process, outcome, and developmental data on infants with HLHS between diagnosis and 12 months of age, 2) improving implementation of consensus standards, tested by teams, into everyday practice across pediatric cardiology centers, and 3) engaging parents as partners in improving care and outcomes. We utilize a quality improvement methodology, known as the adapted learning collaborative model, which expedites the implementation of tools and strategies that facilitate changes such as systematic care coordination, cardiovascular monitoring, and nutritional monitoring into every day practice. The NPC-QIC registry is used to document the impact of these changes on various care processes and outcomes (e.g., mortality rate, readmissions, and weight gain).

Intacs® Corneal Implants is a FDA Humanitarian Use Device (HUD) designed for the reduction or elimination of myopia and astigmatism in patients with keratoconus.

The purpose of this study is to create and maintain a registry, which is a database (a searchable collection of information) about children, adolescents and young adults with pediatric onset of rheumatic diseases. This data may help in the evaluation of the safety and benefit of medications that are prescribed to patients who have rheumatic diseases.

This study is for patients that have been diagnosed with acute myelogenous leukemia (AML) . The investigational drug in this study is AG-120. The purpose of this study is to identify and test the highest dose of AG-120 that can be given safely. Participants can expect to be in the study for as long as the study doctor feels it is in the participants best interest.

Reflexes are important parts of our movements. When reflexes are not working well, movements are clumsy or even impossible. After stroke, reflex responses may change. Researchers have found that people can learn to increase or decrease a reflex response with training. Recently, we have found that rats and people with partial spinal cord injuries can walk better after they are trained to change a spinal cord reflex. Thus, learning to change a reflex response may help people recover after a nervous system injury. In this study, we aim to examine whether learning to change a spinal reflex through operant conditioning training can improve movement function recovery in people after stroke or other damage to the nervous system.

With this research study, we want to learn if methylphenidate (Ritalin® and others) helps people with Alzheimer's disease and apathy (lack of interest or concern for things that others find moving or exciting).

The primary objective of Enroll-HD is to develop a comprehensive repository of prospective and systematically collected clinical research data (demography, clinical features, family history, genetic characteristics) and biological specimens (blood) from individuals with manifest HD, unaffected individuals known to carry the HD mutation or at risk of carrying the HD mutation, and control research participants (e.g., spouses, siblings or offspring of HD mutation carriers known not to carry the HD mutation). Enroll-HD is conceived as a broad-based and long-term project to maximize the efficiencies of non-clinical research and participation in clinical research while ensuring privacy and protections for consenting research participants.

Reflexes are important parts of our movements. When reflexes are not working well, movements are clumsy or even impossible. Researchers have found that people can learn to increase or decrease a reflex response with training. Recently, we have found that rats with spinal cord injuries can walk better after they are trained to change a spinal cord reflex. Thus, learning to change a reflex response may help people recover after a nervous system injury. We are currently studying effects of spinal cord reflex training (e.g., a knee jerk reflex) in people in early adulthood. We hope that the results of this study will help us develop spinal reflex training as a new treatment to help people in early adulthood recover better after spinal cord injury or other damage to the nervous system.

Individuals are being asked to volunteer for a research study. Research studies are voluntary and include only those who choose to take part. The purpose of this research study is to compile a repository of participants who are willing to participate in rehabilitation research studies.

Participants will complete a datasheet that includes contact information and basic medical conditions. Once the datasheet is complete, participants will then await contact from VA Investigators. The study will continue until the participant decides they no longer wish to participate or becomes deceased.

There will be no direct benefit to participants from taking part in this study. The risk associated with this study is a potential loss of confidentiality.

Spinal reflexes take important part in our movement. After spinal cord injury (SCI), reflexes often change. For many years, researchers and doctors have assumed that abnormally acting spinal reflexes lead to movement problems, without clear scientific evidence. For example, in people who suffer spasticity, a common problem after SCI, walking is disturbed, presumably because stretch reflexes (e.g., knee jerk reflex) and some other reflexes are not working well. Yet, which reflex is causing a problem in what way has not been well understood. Such understanding is very important in developing and applying effective therapies for improving gait recovery after SCI. Therefore, in this project, we are studying spinal stretch reflexes and other reflexes during walking, to understand how these reflexes contribute to spastic gait problems in people with chronic incomplete SCI. Successful completion of this project will result in better understanding of spastic gait problems, which in turn, will help us develop more effective therapy application and improve the quality of life in people after SCI.