News

November 20, 2020November 20, 2020

Manufacturing Deficiencies Delay FDA Decision on Sutimlimab for Adults

News

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 ... Read more

November 18, 2020November 18, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

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 ... Read more

November 13, 2020November 13, 2020

Poor Prognosis for Lymphoma-associated AIHA, Case Report Shows

News

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 ... Read more

November 6, 2020November 6, 2020

Apellis, Sobi Partner on Systemic Pegcetacoplan for CAD, Other Rare Diseases

News

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, ... Read more

November 4, 2020November 4, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

News, syndicated

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 ... Read more

October 30, 2020October 27, 2020

UK Organization ’Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

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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 ... Read more

October 23, 2020October 23, 2020

CAD, Other Hemolytic Anemias Increased in Denmark for Past 4 Decades, Study Finds

News

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 ... Read more

October 21, 2020October 21, 2020

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

News, syndicated

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 ... Read more

October 16, 2020October 19, 2020

Infant CAD May Be Treated With IV Immunoglobulin, Case Report Suggests

News

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 ... Read more

October 9, 2020October 9, 2020

Secondary CAD May Cause Complications in Cases of Severe COVID-19, Report Suggests

News

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. ... Read more