A newly discovered biomarker associated with a rare metabolic disorder may facilitate better diagnosis and identification of new drugs for clinical trials for the disease, according to researchers in the Perelman School of ...
Aug 7, 2017
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The Food and Drug Administration approved 41 first-of-a-kind drugs in 2014, including a record number of medicines for rare diseases, pushing the agency's annual tally of drug approvals to its highest level in 18 years.
Jan 2, 2015
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UWA researchers have helped identify the most common cause of congenital lactic acidosis in Roma patients, which could have wide-ranging implications in Australian settings.
Aug 18, 2014
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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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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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Parents must be considered when states decide to expand genetic screening programs for newborns, according to a new study that looked at mandatory testing panels and political pressure by advocacy groups.
Jun 21, 2012
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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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