The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…
Treatment with the anti-cancer immune checkpoint inhibitor Tecentriq (atezolizumab) can cause cold agglutinin disease (CAD), a case report shows. The case was described in a letter to the editor, “Cold agglutinin disease as a new immune-related adverse event associated with anti-PD-L1s…
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…
The profile of patients with mixed autoimmune hemolytic anemia (MAIHA) must be carefully analyzed to facilitate diagnosis and disease management, a study says. The findings of the study, “Immunohematological and clinical characterizations of mixed autoimmune hemolytic anemia,” were published in the Asian Journal of Transfusion Science.
APL-2, Apellis Pharmaceuticals‘ investigational compound for the treatment of autoimmune hemolytic anemia (AIHA) and other complement system diseases, has received the designation of orphan drug from the U.S. Food and Drug Administration (FDA) for the treatment of AIHA. “This orphan drug designation represents an important recognition by…
A new case report described a man with altered body distribution of red blood cells (RBCs), which could have been caused by cold agglutinin disease (CAD). The study, “Abnormal Biodistribution of 99mTc Red Blood Cell Labeled Multi-Gated Acquisition Scan in the Presence of Suspected Cold Agglutinin Disease,”…
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…
A case of simultaneous cryoglobulinemic vasculitis and cold agglutinin disease (CAD) was reported for the first time in a patient, according to Chinese researchers. The rare occurrence was described in the study, “Concomitant cryoglobulinemic vasculitis and cold agglutinin disease successfully treated with bortezomib: A case report,”…
Treatment with low-dose Rituxan can effectively treat patients with warm autoimmune hemolytic anemia (wAIHA) but higher doses may be more beneficial for patients with cold agglutinin disease (CAD), results from a 10-year retrospective study suggest. The study, “Low dose rituximab in autoimmune hemolytic anemia: ten years…
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