Treatment with tofacitinib is well tolerated and effectively induces remission in people with ANCA-associated vasculitis (AAV), while enabling patients to lower their glucocorticoid doses, a small pilot study suggests. These results indicate that tofacitinib may represent a treatment option for active AAV. However, a larger and randomized controlled trial is needed…
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A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…
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…
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…
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…
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,…
Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…
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