CHOC clinicians recently administered the first ever in-human dosing of gene therapy for Hurler Syndrome, a rare lysosomal storage disease.
From the Nursing Frontline of the Batten Disease Battle
CHOC recently hit its second anniversary of the first Brineura infusion, a novel treatment for CLN2 disease, or late infantile Batten disease.
CHOC at forefront of treating Batten disease
Bringing hope to patients and families, CHOC is among a few hospitals in the country to offer treatment for Late Infantile Batten disease.
CHOC Metabolic Specialist Profiled in Science Magazine
The work of a CHOC metabolic disorders specialist and is highlighted in a recent Science magazine article. The article hinges on Dr. Raymond Wang’s work to help a patient with a rare condition called Niemann-Pic Type C, a condition that causes cholesterol to accumulate in the brain, lungs, liver and spleen, leading to deterioration and […]
Meet CHOC’s Specialists: Metabolic Disorders
CHOC Metabolic Disorders Division specializes in diagnosing rare conditions that require hours of unraveling symptoms.
