The U.S. Food and Drug Administration (FDA) has issued a complete response to Sanofi’s application requesting that its investigational therapy sutimlimab be approved for treating hemolysis — the breakdown of red blood cells — in adults with cold agglutinin disease (CAD), the company announced in…
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…
People with non-Hodgkin’s lymphoma (NHL) — a type of blood cancer — that co-occurs with autoimmune hemolytic anemia (AIHA) have a poor prognosis, suggesting a need for further research into this rare combination of conditions, a case report highlights. The case was reported at the 2020 Lymphoma,…
Apellis Pharmaceuticals and Swedish Orphan Biovitrum (Sobi) are collaborating to advance the development of systemic pegcetacoplan, or APL-2, in the treatment of cold agglutinin disease (CAD) and other disorders, the companies announced in a press release. Pegcetacoplan is an investigational therapy designed to limit activation of…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
The incidence and prevalence of most acquired hemolytic disorders, including cold agglutinin disease (CAD), have been rising in Denmark in the past decades, regardless of age and sex, a retrospective study including 36 years of data found. Incidence is the proportion of new cases over a…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
The case of an infant who developed cold agglutinin disease (CAD) shortly after being vaccinated suggests that intravenous immunoglobulin may be an effective treatment for young children with the autoimmune disorder, according to a new report. The report, “Cold agglutinin disease in an infant: remission after intravenous…
Cold agglutinin disease (CAD) may develop as a result of severe COVID-19, further complicating laboratory tests and treatment for these individuals, according to a case report in two patients. The case study, “Cold agglutinin syndrome as a complication of Covid-19 in two cases,” was published…
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