U.S. health officials on Tuesday approved the nation's first gene therapy for an inherited disease, a treatment that improves the sight of patients with a rare form of blindness. It marks another major advance for the emerging ...
Dec 19, 2017
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Four years ago, Janelly Martinez-Amador was confined to a bed, unable to move even an arm or lift her head. At age 3, the fragile toddler had the gross motor skills of a newborn and a ventilator kept her alive.
Apr 24, 2013
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A long-term study of children with a complex heart condition called hypertrophic cardiomyopathy (HCM) found that the risk of death or need for immediate listing for heart transplantation was greatest for those who developed ...
Sep 3, 2013
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Horizon Pharma will spend about $800 million to buy Raptor Pharmaceutical and expand its portfolio of rare disease treatments, a growing area of focus for drugmakers.
Sep 12, 2016
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"Childhood" and "dementia" are two words we wish we didn't have to use together. But sadly, around 1,400 Australian children and young people live with currently untreatable childhood dementia.
Apr 26, 2024
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