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February 26, 2020February 26, 2020

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

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Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the complex process in its Feb. ... Read more

February 24, 2020February 24, 2020

Advances in Diagnosis, Disease Characterization Needed in Canada to Improve aHUS Outcomes, Registry Study Suggests

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Advancements in diagnosis and disease characterization are needed in Canada to improve the clinical outcomes of adults and children with atypical hemolytic uremic syndrome (aHUS), a registry study suggests. ... Read more

February 20, 2020February 21, 2020

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

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An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and ... Read more

February 17, 2020February 17, 2020

Patients Suspected of Having aHUS Should be Treated With Soliris Immediately, Case Study Suggests

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Patients who have symptoms that indicate severe atypical hemolytic uremic syndrome (aHUS) should be diagnosed as quickly as possible so that treatment with Soliris (eculizumab), a potentially life-saving agent, ... Read more

February 10, 2020February 10, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

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The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 10, 2020February 10, 2020

Understanding aHUS-linked Changes in Large Gene Cluster May Help in Diagnosis and Treatment, Review Says

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Understanding the disease-related alterations in a large gene cluster that lead to atypical hemolytic uremic syndrome (aHUS) may help in diagnosing and treating the illness, a recent review suggests. ... Read more

February 5, 2020February 5, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

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The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over ... Read more

February 3, 2020February 3, 2020

Two Missense CD46 Mutations May Lead to Pregnancy-associated aHUS, Case Report Suggests

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Mutations in the CD46 gene — a regulator of the complement system — may lead to pregnancy-associated atypical hemolytic uremic syndrome (aHUS), according to a case study. These findings may help ... Read more

January 27, 2020January 27, 2020

Experts Propose New Diagnostic Criteria to Distinguish TMA Associated With aHUS

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Experts in South Korea have proposed a set of new diagnostic criteria to distinguish thrombotic microangiopathy associated with atypical hemolytic uremic syndrome (aHUS). Their study, “Consensus regarding diagnosis and ... Read more

January 22, 2020January 22, 2020

Soliris Treatment Personalized to Reduce aHUS Patients’ Burden and Medical Costs, Case Study Reveals

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Soliris (eculizumab) maintenance treatment given every three weeks — instead of the standard two weeks — was sufficient to achieve therapeutic blood levels and prevent disease relapse for at least ... Read more

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