Treatment for Dravet Syndrome Zogenix Submits New Drug Application to U.S. Food & Drug Administration for Fintepla for the Treatment of Dravet Syndrome EMERYVILLE, Calif., Feb. 06, 2019 (GLOBE NEWSWIRE) — Zogenix, Inc. (NASDAQ:ZGNX), a global pharmaceutical company developing rare disease therapies, today announced it has completed its rolling submission of a New Drug Application […]Continue Reading ...
Leuven researchers led by professor Bart De Strooper (VIB-KU Leuven) have identified a new role for PARL, a protein linked to Parkinson’s disease. In this week’s edition of the Proceedings of the National Academy of Science, they report that mice lacking PARL instead display specific problems in the nervous system reminiscent of Leigh syndrome. PARL […]Continue Reading ...
With their small size, stubby faces and wide-set eyes, bulldogs, French bulldogs and Boston terriers are among the most popular of domestic dog breeds. Now researchers at the University of California, Davis, School of Veterinary Medicine have found the genetic basis for these dogs’ appearance, and linked it to a rare inherited syndrome in humans. […]Continue Reading ...
An unexpected brain region is important for Dravet Syndrome, finds study As early as 3 months of age, infants with a severe form of epilepsy called Dravet syndrome start having convulsive seizures, during which their arms and legs jerk repeatedly. As they become toddlers, another type of seizure begins to appear. These seizures do not […]Continue Reading ...
When expectant parents learn their child will be born with Down syndrome, they invariably have questions about what this diagnosis will mean for their son or daughter and for the rest of their family. When will their child be able to walk, to speak clearly, to care for most basic needs? Will he or she […]Continue Reading ...
BOSTON, Dec. 13, 2018 (GLOBE NEWSWIRE) — Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company focused on the development and commercialization of therapeutics for the treatment of rare genetic disorders of obesity, today announced the enrollment of the first patient in a pivotal Phase 3 clinical trial evaluating setmelanotide, the company’s first-in-class melanocortin-4 (MC4R) agonist, in […]Continue Reading ...
NEW YORK, Dec. 06, 2018 (GLOBE NEWSWIRE) — Ovid Therapeutics Inc. (NASDAQ:OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced plans to move ahead with a single pivotal Phase 3 trial of OV101 in pediatric patients with Angelman syndrome based on its End-of-Phase 2 […]Continue Reading ...
What are Down syndrome tests? Down syndrome is a disorder that causes intellectual disabilities, distinctive physical features, and various health problems. These may include heart defects, hearing loss, and thyroid disease. Down syndrome is a type of chromosomal disorder. Chromosomes are the parts of your cells that contain your genes. Genes are parts of DNA […]Continue Reading ...
A team of scientists from Sechenov First Moscow State Medical University (MSMU) together with their colleagues described a number of genetic mutations causing Usher syndrome (inherited deaf-blindness) A team of scientists from Sechenov First Moscow State Medical University (MSMU) together with their colleagues from leading scientific centers of Moscow and India described a number of […]Continue Reading ...
Credit: CC0 Public Domain About a quarter of all adults have metabolic syndrome; a syndrome whose most well-known symptoms are obesity, high blood pressure and poor cholesterol levels. Eating differently and exercising more is the general advice for this condition, but that is not the whole story. Ph.D. candidates Yvonne Rozendaal and Fianne Sips developed […]Continue Reading ...
A higher intake of vitamin C is crucial for metabolic syndrome patients trying to halt a potentially deadly cycle of antioxidant disruption and health-related problems, an Oregon State University researcher says. That’s important news for the estimated 35 percent of the U.S. adult population that suffers from the syndrome. “What these findings are really saying […]Continue Reading ...
Leuven researchers led by professor Bart De Strooper (VIB-KU Leuven) have identified a new role for PARL, a protein that has been linked to Parkinson’s disease. In this week’s edition of the Proceedings of the National Academy of Science, they report that mice lacking PARL display specific problems in the nervous system reminiscent of Leigh […]Continue Reading ...
Carpal tunnel syndrome occurs when a nerve in the hand and forearm, known as the median nerve, gets pinched (compressed) within a passage called the carpal tunnel. The carpal tunnel is a narrow canal at the wrist through which the median nerve extends from the forearm to the hand and the first four fingers. It […]Continue Reading ...
November 29, 2018 Audience: Consumer, Health Professional, Pharmacy The U.S. Food and Drug Administration (FDA) is warning that signs and symptoms of a life-threatening side effect called differentiation syndrome are not being recognized in patients receiving the acute myeloid leukemia medicine Idhifa (enasidenib). The Idhifa prescribing information and patient Medication Guide already contain a warning […]Continue Reading ...
Angelman syndrome is a genetic disease with no cure. Children grow up with severe intellectual disabilities and a range of other problems, arguably the worst of which are epileptic seizures. Now scientists at the UNC School of Medicine have found evidence that genetic therapy may prevent the enhanced seizure susceptibility. Published in the Journal of […]Continue Reading ...
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