A 15-month-old girl in Egypt was diagnosed with Gaucher disease after developing severe kidney problems that failed to respond to standard therapies, according to a case report describing what researchers termed a “unique presentation” of Gaucher. While enzyme replacement therapy (ERT) helped ease other Gaucher symptoms for the…
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Our society often rewards the people who are always going, going, going. We measure success by how busy we are, how much we accomplish, and how many people we can please along the way. For a long time, I lived that way, too. I felt the constant pressure to do…
Sharp Therapeutics said it’s arranging a $10 million private investment to advance the development of ‘901, an oral treatment designed to address the neurological and non-neurological symptoms of Gaucher disease. The financing will come through a non-brokered private placement of common stock. STX Partners, Sharp’s largest shareholder, has…
