News

A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases have been selected to receive a Horizon Therapeutics‘ #RAREis Global Advocate Grant. Winners of this year’s inaugural awards are spread across nine countries and represent a total of 29 rare…

A test called Hevylite was superior to standard methods at detecting immunoglobulin M (IgM) — a type of antibody associated with cold agglutinin disease (CAD) — in the blood of people with the disease, a small study has shown. This suggests that Hevylite, sold by Binding Site,…

A man’s cold agglutinin disease (CAD) was caused by a type of blood cancer called B-lymphoblastic leukemia, according to a recent case report. While cancer is a known cause of CAD, this type of cancer was unexpected, the researchers noted, adding that a full work-up is needed to ensure…

Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…

A middle-aged woman’s suddenly black fingertips and dark urine were telltale symptoms that she had developed cold agglutinin disease (CAD), according to scientists. Her case was described by a team of Japanese researchers in the study, “Black fingers and dark urine: cold agglutinin disease,”…

A man with cold agglutinin disease (CAD) successfully underwent microvascular free tissue transfer — a tissue and blood vessel transplant — to repair extensive damage to his face, a case study reported. Authors noted that due to the lack of precedence in the literature, this report provides guidance on…

Enjaymo (sutimlimab) quickly led to increases in hemoglobin and lowered the need for blood transfusions in four women with cold agglutinin disease (CAD), in whom the treatment remained safe and effective for about three years, according to data from a Phase 1 open-label extension trial. Trial data was…

Long-term treatment with Sanofi’s Enjaymo (sutimlimab-jome) safely and effectively prevented red blood cell destruction (hemolysis), anemia, and the need for blood transfusions, while improving the quality of life in adults with primary cold agglutinin disease (CAD). That’s according to 2.5 years of data from the Phase 3…

The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in Cleveland, Ohio. The event, which brings together the rare disease community, will take place at the InterContinental Cleveland Conference…

An elderly woman in Japan is the first reported patient in Asia to develop cold agglutinin disease (CAD) due to COVID-19, according to a new case study. The patient was found to have anemia, or an unusually low red blood cell count, that was not resolved with a blood…