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New targeted therapy options are needed for patients who develop autoimmune hemolytic anemia (AIHA) following a stem cell transplant, a review study says. The study, “Management of refractory autoimmune hemolytic anemia after allogeneic hematopoietic stem cell transplantation: current perspectives,” was published in the Journal of Blood Medicine. AIHA…

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…

Immune rejection and half-matched donors are risk factors for developing autoimmune hemolytic anemia (AIHA) following blood cancer-treating stem cell transplants, a study found. Titled “Autoimmune hemolytic anemia after allogeneic hematopoietic stem cell transplantation in adults: A southern China multicenter experience,” the study was published in the…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

Researchers recently reported the rare case of a 69-year-old Japanese man with paroxysmal atrial fribrillation (PAF) — occasional periods of an irregular, very fast heart rate — who developed hyperkalemia (high potassium levels in the blood) possibly due to cold agglutinin disease (CAD) after undergoing cryoballoon ablation…

A recent case report alerts clinicians to the fact that cold agglutinin disease (CAD) can arise as a complication of Pneumococcal vaccination. This is the first documented case of an adult who developed CAD associated with a specific self-reactive antibody (anti-Pr) following pneumococcal vaccination. The study, “Anti-Pr…