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Long-Term Data With Dawnzera for the Treatment of Hereditary Angioedema

by Madaline Spencer | Dec 16, 2025

Michael Manning, MD, Allergist and Immunologist, discusses long-term data with Dawnzera (donidalorsen) for the treatment of patients with hereditary angioedema (HAE).     HAE is a condition where people have recurrent episodes of severe swelling of the skin...

Effects of Vutrisiran on Cardiac Structure and Function in Patients With Transthyretin Amyloidosis With Cardiomyopathy

by Madaline Spencer | Dec 15, 2025

A recent study, published in Nature Medicine, looked at the effects of vutrisiran on cardiac structure and function in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) through a secondary analysis of the HELIOS-B clinical trial.  ATTR-CM is a rare...

FDA Approves Gene Therapy (Waskyra) for Patients with Wiskott-Aldrich Syndrome

by Madaline Spencer | Dec 12, 2025

The U.S. Food and Drug Administration (FDA) has approved Waskyra (etuvetidigene autotemcel) for the treatment of patients ages 6 months and older with Wiskott-Aldrich syndrome (WAS). WAS is a disease with immunological deficiency and reduced ability to form blood...

Advancing Thyroid Eye Disease Care: Integrating Tepezza, New Technology, and Optimizing Multidisciplinary Teams

by Madaline Spencer | Dec 12, 2025

Maja Kostic, MD, PhD, neuro-ophthalmologist at Bascom Palmer Eye Institute and Assistant Professor of Ophthalmology at the University of North Carolina at Chapel Hill, discusses advancing thyroid eye disease (TED) care.     TED is a rare autoimmune disease...

FDA Adds Marginal Zone Lymphoma to Indication of Breyanzi

by Madaline Spencer | Dec 11, 2025

The U.S Food and Drug Administration (FDA) has approved Breyanzi (lisocabtagene maraleucel; liso-cel) for the treatment of adults with relapsed/refractory marginal zone lymphoma (MZL) who have received at least two prior lines of systemic therapy. MZL is a usually...
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