Group Focuses on Rare Disease Clinical Trial Participation

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

A lower, 100 mg dose of Nucala (mepolizumab) is equally effective at treating eosinophilic granulomatosis with polyangiitis (EGPA) as the standard 300 mg dose, and leads to less frequent adverse events, or AEs, a large European study showed. These findings suggest that a lower monthly dose of Nucala…

Today, I woke up hurting. While it wasn’t the pain that someone with fibromyalgia or rheumatoid arthritis might suffer, it caused enough discomfort that I couldn’t ignore it. A dull ache had settled in, from my neck to my knees, making it difficult to get out of bed. The previous…

A first course of rituximab is sufficient to trigger the development of anti-rituximab antibodies in patients with systemic lupus erythematosus (SLE) but not in those with ANCA-associated vasculitis (AAV), a study found. The researchers had thought that rituximab might have similar effects in patients with SLE and AAV…

I’m so tired. That thought threatens to dominate my mindset. It affects everything, including my outlook, motivation, and productivity. Later in the day, it might even tip my appetite levels out of balance. I just want to rest. I close my eyes again, but my brain is already scrolling through…

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…

Inflammatory vaginitis — inflammation of the vagina — may develop in women receiving long-term treatment with rituximab for ANCA-associated vasculitis (AAV) or other autoimmune diseases, a U.S. study says. The findings suggest that “women treated with rituximab should be screened for symptoms such as vaginal discharge and vaginal pain,” its…

Humans are configured as social animals, regardless of personality type. We rely on each other, not only at a basic level for security, comfort, and good conversation, but also on a higher plane for things like artistic inspiration and love. The necessity of having “our people” around is never…

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…

Mycophenolate mofetil (MMF) is well-tolerated and can be used to induce remission in people with eosinophilic granulomatosis with polyangiitis (EGPA), a type of ANCA-associated vasculitis (AAV), a real-world study shows. Two-thirds of patients with newly diagnosed or relapsing EGPA achieved remission, or had no symptoms appearing for a time,…