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. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

A medication for high blood pressure, Tekturna (aliskiren) may be used as an add-on therapy to manage hypertension associated with atypical hemolytic uremic syndrome (aHUS) until more adequate treatments become available, a case report finds. The study, “Aliskiren as an adjunct therapy for atypical hemolytic uremic…

Atypical hemolytic-uremic syndrome can arise following an aortic dissection — a serious condition in which the inner layer of the aorta tears open — according to a new case report. The study, “Atypical Hemolytic Uremic Syndrome following Acute Type A Aortic Dissection,” was published in the journal…

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) —…

Bexsero, a vaccine developed as a treatment for serogroup B meningococci — the bacteria that can cause meningitis — is only effective in 50% of people with atypical hemolytic uremic syndrome (aHUS), a new study reports. The study, “Low efficacy of vaccination against serogroup…

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…

Scientists have discovered, in a Chinese patient, new mutations in the DGKE gene that can cause atypical hemolytic uremic syndrome (aHUS), a case report shows. These new mutations expand current knowledge of this gene in the context of aHUS, and highlight potential racial differences in DGKE genetic…

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…

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…