News

Cold agglutinin disease (CAD) patients have an increased risk of developing blood clots compared to the general population, a retrospective Danish study shows. The findings of the study, “Occurrence, thromboembolic risk, and mortality in Danish patients with cold agglutinin disease” were published recently in the journal Blood Advances. In…

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…