News

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 the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…

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 study, “Differences in Aerobic Fitness between an Obese Adolescent with Prader-Willi Syndrome and Other Obese Adolescents and Exercise Training…

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 incidence of mental health problems in those with PWS compared to the general population, scientists said. The study, “…

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 study suggests. The study, “Metabolic Parameters in…

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 in this series A funding include the Foundation for Prader Willi Research. “Prader-Willi syndrome, or PWS,…

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 lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

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 #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…

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 6 to 65. The study (NCT04257929) is expected to start enrolling participants soon, the company said in a…

Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, and aims to supports work by researchers and clinicians into a better understanding of and treatments for rare diseases, as well…

Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those  looking to begin the complex process in its Feb. 20 webinar.  William Whitman…