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Custom Gene Editing Saves Infant with Rare Metabolic Disorder
In a groundbreaking first, doctors developed a custom gene- editing therapy in just seven months to treat a baby with a deadly and ultra-rare metabolic disorder.
The Rare360 Editorial Team
8 hours ago2 min read
From CRISPR to Jumping Genes: The Next Breakthrough in Genetic Science
New research on "jumping genes" could lead to safer, more precise gene-editing techniques.
The Rare360 Editorial Team
Oct 3, 20244 min read
Retinitis Pigmentosa: A Guide to Symptoms and Management
Retinitis Pigmentosa (RP) is a group of rare, inherited eye disorders leading to progressive vision loss.
The Rare360 Editorial Team
Sep 23, 20249 min read
FDA Extends Priority Review Period for Kresladi/ RP-L 201 in Treating Severe Leukocyte Adhesion Deficiency-I (LAD-1)
Explore the FDA's extension of the priority review period for Kresladi, a groundbreaking gene therapy for severe LAD-1
The Rare360 Editorial Team
Feb 19, 20242 min read
Milestone in Gene Therapy: Casgevy Approved for Transfusion-Dependent Beta Thalassemia
Explore the FDA's groundbreaking approval of CASGEVYâ„¢ the treatment of transfusion-dependent beta-thalassemia (TDT).
The Rare360 Editorial Team
Jan 18, 20243 min read
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