Health Problem: Gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are a rare and heterogeneous group of neoplasms derived from neuroendocrine cells within the gastrointestinal tract and pancreas, where curative surgery options are limited. GEP-NETs are estimated to affect approximately 3 individuals per 100,000 people per year.
Eculizumab (Soliris) for Treatment of Refractory Generalized Myasthenia Gravis
Health Problem: Myasthenia gravis (MG) is a rare autoimmune disorder that impacts the neuromuscular system, leading to muscle weakness, particularly affecting the nasopharyngeal, limb, and respiratory muscles. MG is a progressive disease caused by autoantibodies that interfere with the transmission of acetylcholine from nerve endings to muscl…
Inhaled Nitric Oxide for the Treatment of Respiratory Failure in Preterm Newborns
Health Problem: Respiratory distress syndrome (RDS) is one of the most common causes of preterm infant respiratory failure and mortality, with RDS occurring in 20,000 to 30,000 newborn infants annually in the United States. RDS results from developmental immaturity of the lungs, which leads to insufficient surfactant production and function. …
Tezacaftor-Ivacaftor (Symdeko) for Treatment of Cystic Fibrosis
Health Problem: Cystic fibrosis (CF) is an autosomal recessive genetic disease due to mutations in the cystic fibrosis transmembrane receptor (CFTR) gene. The basic defect results in poor chloride and bicarbonate transport, resulting in dehydration of secretions in the lungs, pancreas, and other organs. The bronchi and lungs are affected by r…
Lumacaftor-Ivacaftor (Orkambi) for Treatment of Cystic Fibrosis
Health Problem: Cystic fibrosis (CF) is an autosomal recessive genetic disease due to mutations in the cystic fibrosis transmembrane receptor (CFTR) gene. The basic defect results in poor chloride and bicarbonate transport resulting in dehydration of secretions in the lungs, pancreas, and other organs. The bronchi and lungs are affected by re…
Nusinersen (Spinraza; Biogen) for Spinal Muscular Atrophy
Health Problem: Spinal muscular atrophy (SMA) is a recessive genetic disorder characterized by progressive proximal muscle weakness and atrophy. SMA is predominantly caused by deletions or mutations of survival motor neuron (SMN) SMN-1, which leads to degeneration of alpha motor neurons of the spinal cord anterior horn cells. This affects the…
Recombinant Human Growth Hormone Treatment in Adults with Prader-Willi Syndrome
Health Problem: Prader-Willi syndrome (PWS) is a genetic disorder that is highly variable and can affect multiple body systems; there is an associated pattern of physical findings with significant cognitive, neurologic, endocrine, and behavioral abnormalities. Studies in the United States have reported a prevalence rate ranging from 1 per 16,…
Liraglutide (Saxenda) For Weight Loss in Nondiabetic Obese Adults
Health Problem: The prevalence of obesity remains high in the United States. Recent estimates indicate that more than one-third of adults have obesity. Obesity is a serious health concern because it adversely affects mental health and quality of life, and is associated with leading causes of death, including diabetes, heart disease, stroke an…
Atezolizumab (Tecentriq) for Non-Small Cell Lung Cancer
Health Problem: Lung cancer is the second most common form of cancer in men (after prostate cancer) and women (after breast cancer), accounting for approximately 14% of all new cancers. Non-small cell lung cancer (NSCLC) accounts for > 80% of all lung cancer cases, and it includes 2 major types: non-squamous, including adenocarcinoma, large-c…
Glucagon-Like Peptide-1 Receptor Agonists for the Treatment of Obesity in Women with Polycystic Ovary Syndrome
Health Problem: Polycystic ovary syndrome (PCOS) is a common endocrine disorder affecting 6% to 20% of women of reproductive age. It is characterized by oligoovulation (menstrual irregularity), hyperandrogenism (increased testosterone levels), and polycystic ovaries. Obesity is a common feature of PCOS, affecting approximately 40% to 80% of w…