dermatologists

xanthoma

Study Testing Cholesterol-Lowering Drug in ALGS Patients Now Recruiting

A new study testing the safety and efficacy of atorvastatin in treating xanthomas in patients with Alagille syndrome (ALGS) is recruiting participants. The prospective phase 4 study aims to clarify the safety and efficacy of atorvastatin to provide a reference for the treatment of xanthomas in children with the disease. The study will recruit 10…

sad woman

Living With SM Impacts Relationships and Emotional Status Along With Physical Health

Systemic mastocytosis (SM) can have a multifaceted impact on patients’ health, relationships, and emotions, according to a study published in the European Journal of Oncology Nursing. Through purposive sampling and semistructured interviews, the study identified 3 main categories of impacts that SM had on patients’ lives—the persistent presence of the disease, struggling against ignorance, and…

clinical research

Lanadelumab Shown to Provide Safe and Effective Prophylaxis for Hereditary Angioedema

Lanadelumab (Takhzyro®) is a safe and effective prophylactic drug for use in patients with hereditary angioedema, researchers reported in a study published in the World Allergy Organizational Journal. Hereditary angioedema with C1 inhibitor deficiency is characterized by recurring, painful edema attacks involving subcutaneous or submucosal tissue, primarily driven by the overproduction of bradykinin. The disease…

FDA

FDA Accepts New Drug Application for Potential HAE Treatment STAR-0215

The US Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for STAR-0215, an experimental new treatment for hereditary angioedema (HAE). A phase 1a clinical trial of STAR-0215 in healthy volunteers is expected to begin soon, with preliminary results anticipated before the end of the year, according to a press release…

mast cell

New Study Sheds Light on Proinflammatory Mechanism in Systemic Mastocytosis

Altered distribution of leukocyte subsets and a proinflammatory proteome was seen in 2 independent groups of patients with indolent systemic mastocytosis, according to a new study published in the Journal of Allergy and Clinical Immunology. This observation led the authors of the study to hypothesize that neoplastic mast cells could be recruiting and activating immune…

female patient and doctor

More Knowledge About SM May Improve Patients’ Health-Related Quality of Life

Systemic mastocytosis and mast cell activation syndrome both have a significant negative impact on patients’ health-related quality of life, a new study published in the Orphanet Journal of Rare Diseases found. However, the level of information patients receive about their disease seems to have a direct impact. “This is not only an issue in rare…

clinical trial data

New Trial Testing Lanadelumab for HAE Now Open

A new clinical trial testing the effect of lanadelumab in teenagers and adults with hereditary angioedema (HAE) is now open.  The observational study will compare the rate of HAE attacks that require on-demand treatment before and within 2 years of lanadelumab treatment. The trial, sponsored by Takeda aims to recruit an estimated 50 participants at…

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