News

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…

Treatment with Soliris (eculizumab) during pregnancy may help prevent subsequent relapses of pregnancy-associated atypical hemolytic uremic syndrome (aHUS), according to a case report. The report, “Eculizumab Maintenance and the Prevention of Atypical Hemolytic Uremic Syndrome Relapse During Pregnancy: A Case Report,” was published in the…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

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…