National Institutes of Health NIH Office of Rare Diseases Research. Web site aims to answer questions about rare diseases and the activities of the ORDR for patients, their families, healthcare providers, researchers, educators, students, and anyone with concern for and interest in rare diseases. The site provides information about ORDR-sponsored biomedical research, scientific conferences, and rare and genetic diseases. It also serves as a portal to information on major topics of interest to the rare diseases community.
National Institute of Arthritis and Musculoskeletal and Skin Disease NIAMS - The mission of the National Institute of Arthritis and Musculoskeletal and Skin Diseases is to support research into the causes, treatment, and prevention of arthritis and musculoskeletal and skin diseases; the training of basic and clinical scientists to carry out this research; and the dissemination of information on research progress in these diseases.
Disease organization representing children and families who are affected by a pediatric neurotransmitter disease. Helps children and families who are affected by PND's, support the identification of new PND's, find better treatments and ultimately a cure for those diseases that are already known. The PND Association is a non-profit, voluntary organization
CDC - Centers for Disease control and Prevention - Your Online Source for Credible Health Information. The CDC is recognized around the world as a leading force in public health expertise.
National Digestive Diseases Information Clearinghouse (NDDIC) A service of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), NIH Heartburn, Gastroestophageal Reflux Disease (GERD)
The National Association of Chronic Disease Directors (NACDD) is a public health association for chronic disease program directors of each state and U.S. territory. Since its founding in 1988, NACDD has been a national leader in the effort to reduce chronic diseases by mobilizing its members to advocate for preventive policies and programs, encourage knowledge sharing and develop model rtnerships for health promotion through state and community-based prevention strategies
A Pilot Study to Assess the Efficacy and Effectiveness of High-Frequency Chest Wall Oscillation (HFCWO) in Preventing Pulmonary Exacerbations in Children With Muscle Weakness and Restrictive Lung Disease To further demonstrate that airway clearance provided by Vest therapy reduces the frequency of respiratory exacerbations requiring hospitalization or antibiotic utilization in patients with muscle weakness and
restrictive lung disease.
National Heart Lung and Blood Institute (NHLBI) Diseases and Conditions Index (DCI). This Web-based health index gives you a quick and easy way to get complete and dependable information about heart, lung, and blood diseases and sleep disorders.
A preeminent non-profit professional association of pediatric neurologists in the United States, Canada, and worldwide devoted to fostering the discipline of child neurology and promoting the optimal care and welfare of children with neurological and neurodevelopmental disorders. These disorders include epilepsy, cerebral palsy, mental retardation, learning disabilities, complex metabolic diseases, nerve and muscle diseases and a host of other highly challenging conditions
NICHD National Institute of Health Eunice Kennedy Shriver National Institute of Child Health & Human Development - Center for Research for Mothers & Children CRMC - CRMC serves as a principal source of NIH support for research and research training in maternal and child health. Supported scientists are advancing fundamental and clinical knowledge concerning maternal health and child development problems, such as gestational diabetes, antecedents of adult diseases, obesity and overweight, specific learning disabilities, mechanisms of cognition and learning, growth retardation, HIV/AIDS, and other congenital infections and diseases. The Center also houses the primary federal research entity for research to understand the effects and effectiveness of pharmaceuticals on maternal and child health, as well as a large portfolio on the unique aspects of HIV/AIDS in women and mothers.
Federation of individuals and organizations working together to build a better world for people affected by rare diseases. given a top (4-star) rating by a leading independent evaluator of nonprofit organizations
Pittsburgh Development Center of Magee-Womens Research Institute explores the molecular biology of cell function-- in gametes, embryos, stem cells, maternal/fetal efficacy of assisted reproduction technologies, the origins of developmental diseases, the causes and prevention of adverse pregnancy outcomes and the potential of stem cells for treating human disease
Study conducted and completed to evaluate the efficacy, acceptability, and safety of Axid Oral Solution versus placebo in the treatment of gastroesophageal reflux disease (GERD) in infants age 30 days up to 1 year.
Study is to evaluate the pharmacokinetics (blood levels), pharmacodynamics (how the drug acts on the body), and the safety of rabeprazole sodium for the treatment of gastroesophageal reflux disease (GERD) in new borns and pre-term infants (less than 44 weeks of age).
The Rare Diseases Clinical Research Network (RDCRN) is made up of 19 distinctive consortia that are working in concert to improve availability of rare disease information, treatment, clinical studies, and general awareness for both patients and the medical community. The RDCRN also aims to provide up-to-date information for patients and to assist in connecting patients with advocacy groups, expert doctors, and clinical research opportunities
Pub Med Article Abstract presents a short clinical review of metabolic diseases associated with metabolic acidosis in the newborn and discusses appropriate investigations and
differential diagnoses. It is important to remember the possibility of metabolic diseases as the cause of metabolic acidosis in the newborn period.