News

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Group Focuses on Rare Disease Clinical Trial Participation

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

COVID-19 May Trigger CAD in Children

SARS-CoV-2, the virus that causes COVID-19, may trigger cold agglutinin disease (CAD) in children, a case report suggests. The report, “Autoimmune hemolytic anemia in children with COVID-19,” was published in the journal Pediatric Blood & Cancer. Autoimmune hemolytic anemia (AIHA), an autoimmune disease characterized by…

Yellow Tongue Leads to Rare CAD Diagnosis

A young boy’s bright yellow tongue led doctors to a diagnosis of cold agglutinin disease (CAD), as described in a report of this rare case. The report, “An Icteric Tongue,” was published in The New England Journal of Medicine in the series Images in Clinical Medicine.

Register Now for Global Genes’ RARE Patient Advocacy Summit

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…