News

Russia has approved for patient use the first biosimilar of Soliris (eculizumab), a monoclonal antibody for the treatment of atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH). The product was created by the…

Hypertensive emergency and the presence of rare complement mutations are essential to estimate the prognosis of kidney disease in patients with atypical hemolytic uremic syndrome (aHUS). The study, “Impact Of Hypertensive Emergency And Complement Rare Variants On Presentation And Outcome Of Atypical Hemolytic Uremic Syndrome,” was published in…

A novel blood test may help monitor disease activity in patients with atypical hemolytic uremic syndrome (aHUS), as well as those at risk for relapse, a preliminary study reports. The test measures the ability of a person’s serum to activate complement factor C5b-9, which is usually higher in…

New research shows how an antibody was able to enhance the function of a member of the complement system, called complement factor H (FH), responsible for keeping this system in check. Augmenting FH with the newly discovered antibody reduced red blood cell burst in blood samples of atypical hemolytic uremic…

A rare case of atypical hemolytic uremic syndrome (aHUS) manifesting in generalized seizures in a 14-month-old girl highlights the importance of prompt diagnosis and treatment to ensure better outcomes for this young population, a report says. The case was described in a study, “…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bathdissertation, she analyzed Brexit’s long-term impact…

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 to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

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 to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…

Omeros Corporation’s lead therapy candidate, OMS721, being developed for the treatment of complement- and inflammation-related disorders including atypical hemolytic uremic syndrome (aHUS), will now be known as narsoplimab, the company announced. The generic name narsoplimab was approved by the United States Adopted Names (USAN) Council in consultation…

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 already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…