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Suppressing the pro-inflammatory complement pathway using sutimlimab, in addition to other measures, can help prevent red blood cell destruction in patients with cold agglutinin disease (CAD) undergoing major surgery, a recent case report suggests The study, “Sutimlimab, an investigational C1s inhibitor, effectively prevents exacerbation of hemolytic anaemia…
Sutimlimab increased hemoglobin, lowered levels of red blood cell destruction markers, and eased fatigue and symptoms related to anemia in patients with cold agglutinin disease (CAD), according to a recent analysis of CADENZA. In…
A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than for those without rare diseases. This study provides evidence of the potential effect rare diseases may have on public health…
Cold agglutinin disease (CAD) in an elder woman worsened an underlying autoimmune condition that subsequently led to acute liver failure, a case study reports. This case highlights a need for informed understanding of CAD and its associations with other autoimmune processes, and how it can lead to early interventions…
Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…
The virus that causes COVID-19, SARS-CoV-2, may have triggered both warm and cold autoimmune hemolytic anemia (AIHA) in a 51-year-old man, a case study reports. The case report, “Warm and Cold Autoimmune Hemolytic Anemia in the Setting of COVID-19 Disease,” was published in the journal Cureus. AIHA comprises a…
The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…
A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.
A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…
In some cases, vaccines for COVID-19 can lead to a disease flare in people with cold agglutinin disease (CAD) and related conditions, but these flares can be readily managed with proper treatment, according to an Italian study. These findings point to a need for close monitoring when these patients…
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