News

Patients with cold agglutinin disease (CAD) are twice as likely to die of their disease within the first five years after being diagnosed, when compared with the general population, a study says. The findings announced by Bioverativ — a pharmaceutical company now part of Sanofi that is focused…

Treatment with the anti-PD-1 targeted checkpoint inhibitor Keytruda (pembrolizumab) was found to trigger autoimmune hemolytic anemia (AIHA) in a man with non-small cell lung cancer (NSCLC). The case was described in the study, “Pembrolizumab-induced Autoimmune Hemolytic Anemia and Hemophagocytic Lymphohistiocytosis in Non-small Cell Lung…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bath dissertation, she analyzed Brexit’s long-term impact…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…

Apellis Pharmaceuticals and SFJ Pharmaceuticals are now collaborating on the development of investigational therapy APL-2 as a treatment for cold agglutinin disease (CAD) and warm antibody hemolytic anemia (wAIHA). This follows the announcement of a newly established partnership to support the clinical program of APL-2 for the treatment…

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…