News

As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…

In patients with ANCA-associated vasculitis (AAV), the reappearance of ANCA autoantibodies may work as a biomarker for predicting relapse, a new study suggests. Routine monitoring of ANCA antibody levels should be implemented in AAV patients in remission as a means to determine their risk of relapse, according to Japanese…

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…

Researchers have reported the case of a patient who developed ANCA-associated vasculitis (AAV) after taking a thyroid medication for Graves’ disease. While this medication has been linked to AAV in other studies, this was the first time the condition manifested as a perforation of the nose septum, Japanese researchers said.

The protein myeloperoxidase (MPO) may increase the activation of neutrophils — key immune cells in ANCA-associated vasculitis — and heighten vascular injury in patients, a new study shows. The study, “Myeloperoxidase influences the complement regulatory activity of complement factor H,” was published in the journal Rheumatology. ANCA-associated vasculitis (AAV)…

Patients with end-stage renal disease due to granulomatosis with polyangiitis — a type of ANCA-associated vasculitis — live significantly longer after receiving a kidney transplant, a nationwide review study shows. This was mainly due to a 90% reduction in the risk for cardiovascular death, researchers found. The findings suggest that promptly…

Interstitial lung disease — a group of conditions characterized by scarring of the lungs — may develop in patients with all three subtypes of ANCA-associated vasculitis (AAV), a retrospective study shows. The study, “Interstitial Lung Disease in ANCA-Positive Vasculitis Patients,” will be presented at the American…

A new case study supports previous findings that hydralazine — a medication used to treat high blood pressure — may cause ANCA-associated vasculitis with severe kidney injury, leading its researchers to recommend its use “generally be avoided.” A separate case report, published in March, linked this medication to end-stage kidney disease…

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…