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Rare Care Podcast

Browse Rare Disease Advisor’s engaging “Rare Care” podcast series, featuring interviews with leading researchers, stakeholders, and influencers within the rare disease community.

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Hemophilia Therapies

What We Know About Efmoroctocog Alfa in Hemophilia A

There has been a number of clinical studies on one of the newer drugs on the market for hemophilia A: efmoroctocog alfa. It has generated excitement among the medical community because it is not a traditional intravenous (IV) replacement therapy; instead, it is a prophylactic medication and has a longer half-life than conventional factor VIII…

Examining the Relationship Between LAL-D and Skeletal Homeostasis

Lysosomal acid lipase deficiency (LAL-D) is a rare disease that results in ectopic lipid accumulation in the body, especially in the liver, spleen, small intestine, blood, and adrenal glands. Given that unesterified cholesterol and fatty acids play an important role in bone metabolism, a team of scientists has proposed that LAL-D also significantly disrupts skeletal…

liver and bile duct

Visualizing Bile Duct Abnormalities in Alagille Syndrome in 3D

A few centuries ago, if a patient complained of general maladies such as “fever” or “chest pain,” the physician could only guess at what was wrong and provide symptomatic relief (such as sponging down the fever and ensuring that the patient is eating and ambulating). We should not be too quick to dismiss the “guesses”…

Cold Agglutinin Disease Therapies

Clinical Thinking in Diagnosing and Managing Cold Agglutinin Disease

Sigbjørn Berentsen, MD, PhD, is a consultant hematologist and senior researcher in the Department of Research and Innovation at Haugesund Hospital in Norway. He has authored a number of studies on autoimmune hemolytic anemia (AIHA), and its cold agglutinin disease (CAD) subtype. He is a respected expert on the subject in a field in which…

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