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NORD welcomes advance in US Congress of key rare disease legislation

The National Organization for Rare Disorders (NORD) welcomes the advancement, by the U.S. House of Representative’s Energy and Commerce Health Subcommittee, of “critical” bills that encourage rare disease therapy development and improve patient access to care. The nonprofit organization, which supports rare disease patients through advocacy, research funding, education,…

aHUS disease in adolescence linked to permanent kidney function loss in study

Atypical hemolytic uremic syndrome (aHUS) starting during adolescence is associated with more permanent and severe kidney dysfunction when compared with infantile-onset disease, a study from Turkey reported. “In the long term, adolescents may have significant permanent loss of [kidney] functions and higher [end-stage kidney disease] rates,” the researchers wrote.

Soliris may induce remission of aHUS in children with anti-FH antibodies

Soliris (eculizumab) can effectively induce and maintain remission of atypical hemolytic uremic syndrome (aHUS) in children with autoantibodies that target complement factor H (FH). That’s according to a study that found the therapy reduced anti-FH antibodies no matter the type of immunosuppressive treatments used with it. They also…

Soliris effectively treated aHUS in young woman with SLE: Case report

Soliris (eculizumab) was used to effectively treat atypical hemolytic uremic syndrome (aHUS) in a young woman with systemic lupus erythematosus (SLE), another autoimmune condition, a case report shows. The therapy was safely discontinued without causing an aHUS relapse, while immunosuppressant treatments for SLE were maintained. This “case highlights…

Woman had aHUS, kidney failure after COVID-19: Case study

A 78-year-old woman developed atypical hemolytic uremic syndrome (aHUS) and acute kidney failure after contracting an infection with SARS-CoV-2, the virus that causes COVID-19, doctors wrote in a case study. Diffuse alveolar hemorrhage, a condition characterized by bleeding from the lung’s alveoli, or the tiny air sacs where gas…