News

March 27, 2020March 27, 2020

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

News

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. ... Read more

March 24, 2020March 24, 2020

PWS-associated Gene Involved in Integration of Vision and Motor Control in the Brain

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Altered gene expression underlies impaired function of brain areas regulating vision and motor control, which may be associated with behavioral issues in people with Prader-Willi syndrome (PWS) and other ... Read more

March 23, 2020March 23, 2020

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

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Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see ... Read more

March 19, 2020March 19, 2020

Tailored Exercise May Benefit Obese PWS Patients, Case Study Suggests

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Obese people with Prader-Willi syndrome (PWS) should have access to exercise training programs different from those of obese people not affected by PWS, a case report suggests. The case ... Read more

March 17, 2020March 17, 2020

Variants in SNORD116 Gene Linked with PWS, Paranoia in Women, Study Finds

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Small mutations in SNORD1116, a gene previously linked with typical manifestations of Prader-Willi syndrome (PWS), is associated with paranoia in women, a study shows. This finding reflects the greater ... Read more

March 12, 2020March 12, 2020

Higher Insulin Resistance Linked to Mental Illness in PWS, Study Suggests

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Children with Prader-Willi syndrome (PWS) may be particularly prone to metabolic alterations, including greater insulin resistance, which may be linked to increased risk for mental problems such as psychosis, a ... Read more

March 10, 2020March 10, 2020

Lipidio Completes First Financing Step to Develop PWS Therapy GDD3898

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Lipidio Pharmaceuticals has completed the first step of financing to support its development of GDD3898, a treatment candidate for Prader Willi syndrome (PWS) and other conditions associated with obesity. Investors ... Read more

March 9, 2020March 9, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

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The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more

March 5, 2020March 6, 2020

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

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In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the ... Read more

March 5, 2020March 5, 2020

Phase 2 Trial Launched to Test Pitolisant for Excessive Sleepiness in PWS

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Harmony Biosciences has kicked off a Phase 2 clinical trial to evaluate the safety and effectiveness of pitolisant to treat excessive daytime sleepiness in people with Prader-Willi syndrome, ages ... Read more

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