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March 14, 2019March 14, 2019

ConSynance Therapeutics Will Participate in Prader-Willi Trial Consortium

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ConSynance Therapeutics, a biotechnology company that has developed two new candidate medicines for Prader-Willi syndrome (PWS), announced that it has joined the PWS Clinical Trial Consortium. The PWS Clinical Trial ... Read more

March 12, 2019March 12, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

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With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed ... Read more

March 5, 2019

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

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Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known ... Read more

February 28, 2019February 28, 2019

Tesomet Safe for Teens with PWS; More Data Needed to Show Efficacy

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Data from an ongoing Phase 2a clinical trial showed that Tesomet (tesofensine/metoprolol) is safe and well-tolerated by adolescents with Prader-Willi syndrome (PWS). Eight of the nine adolescents included in ... Read more

February 26, 2019February 26, 2019

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

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The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have ... Read more

February 21, 2019February 21, 2019

Vienna to Host RARE2019 Meeting on Rare Diseases

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About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The ... Read more

February 19, 2019February 19, 2019

3-month Training Program Improves Hand Function in PWS Patients, Study Reports

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A low-intensity training program improved hand function in adults with Prader-Willi syndrome (PWS), according to results of a pilot study in six patients. The research, “Hand strength and dexterity ... Read more

February 14, 2019February 14, 2019

Bariatric Surgery a Feasible Option for Adolescents with Prader-Willi, Case Report Suggests

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With careful multidisciplinary evaluation and psychosocial support, adolescents with Prader-Willi syndrome (PWS) with severe obesity can undergo bariatric surgery with successful results, a case report suggests. The study, “Laparoscopic sleeve ... Read more

February 11, 2019February 11, 2019

Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

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Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run ... Read more

February 7, 2019February 7, 2019

Food-related Behavior in PWS May be Linked to Neurological Response

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Involuntary reflexes such as being startled are abnormal in Prader-Willi Syndrome (PWS) patients, and lower startle response to food images may explain their eating habits, researchers from the University of ... Read more

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