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Clinical Staging of Hypertrophic Cardiomyopathy in Fabry Disease

by Madaline Spencer | Apr 22, 2026

A retrospective observational study published in the International Journal of Cardiology evaluated a new clinical staging of hypertrophic cardiomyopathy in Fabry disease. Fabry disease is a lysosomal disorder caused by mutations in the GLA gene. It is characterized by...

Phase 3 Results of Brepocitinib for the Treatment of Dermatomyositis

by Madaline Spencer | Apr 21, 2026

Ben Zimmer, CEO of Priovant Therapeutics, discusses results from the recent phase 3 study testing brepocitinib for the treatment of dermatomyositis (DM).     DM is a rare autoimmune condition that causes skin changes and muscle weakness. Symptoms can include...

Tepezza (Teprotumumab) for the Treatment of Thyroid Eye Disease

by Madaline Spencer | Apr 20, 2026

Prem Subramanian, MD, PhD, Chief of Neuro-Ophthalmology at the University of Colorado Anschutz, discusses Tepezza (teprotumumab-trbw) for the treatment of thyroid eye disease (TED).     TED is a chronic endocrine, autoimmune disease characterized by...

Arms Wide Open Childhood Cancer Foundation and CureFest

by Madaline Spencer | Apr 17, 2026

Dena Sherwood, mother to a neuroblastoma survivor and Founder of Arms Wide Open Childhood Cancer Foundation, discusses her organization and the CureFest event.     Neuroblastoma is a rare childhood cancer, but it is the most common extracranial solid tumor...

FDA Approves Sparsentan to Treat Focal Segmental Glomerulosclerosis

by Madaline Spencer | Apr 16, 2026

The US Food and Drug Administration (FDA) has approved Filspari (sparsentan) to reduce proteinuria in patients with focal segmental glomerulosclerosis (FSGS). The indication includes adult and pediatric patients ages 8 years and older without nephrotic syndrome. In...
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