According to the Wall Street Journal, the National Institutes of Health (NIH) has reversed its initial decision to prevent two of its researchers from meeting with federal investigators to comment…
Noonan Syndrome Noonan syndrome (NS) is a rare disorder that is typically diagnosed at birth. It affects the child's development resulting in stunted growth, abnormal facial features, and heart defects,…
At the 2019 annual meeting of The Endocrine Society, ENDO 2019, results from a recent study evaluating burosumab for the treatment of X-linked hypophosphatemia (XLH) were presented. This drug was…
Kennedy disease or, Spinal and Bulbar Muscular Atrophy (SBMA) is a form of Spinal Muscular Atrophy (SMA). SBMA causes muscle weakness throughout the limbs which results in impaired mobility (typical…
According to a story from Market Screener, the biopharmaceutical company Marinus Pharmaceuticals, Inc. recently announced its plans to initiate a Phase 3 study that is intended to test ganaxolone as…
What is Bronchopulmonary Dysplasia? Bronchopulmonary Dysplasia (BPD) is a diagnosis most commonly given to premature newborns. In children, BPD is also the most common cause of Pulmonary Hypertension (PH). Premature babies often develop…
Multiple Sclerosis (MS) was long believed to be an illness that could only affect adults. In fact, the pediatric form of this disease was not discovered until just over 10…
New Research to be Presented In just a few days the American College of Cardiology will host their 68th Annual Scientific Session (ACC.19). It will be held in New Orleans,…
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