The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder.
Oct 30, 2023
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(HealthDay)—Ravicti (glycerol phenylbutyrate) has been approved by the U.S. Food and Drug Administration to treat certain urea cycle disorders (UCDs) in people 2 years and older.
Feb 1, 2013
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Enzyme therapy proves effective in treating LSDs, whilst gene therapy is an upcoming contender.
Apr 15, 2014
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Independent US presidential candidate Robert F. Kennedy Jr's health is in the spotlight again—this time relating to a neurological issue.
May 13, 2024
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One of the most common types of brain tumors in adults, glioblastoma multiforme, is one of the most devastating. Even with recent advances in surgery, radiation and chemotherapy, the aggressive and invasive tumors become ...
Jan 17, 2013
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University of Adelaide research using special adult stem cells is promising new hope for better treatments for the devastating genetic disease Hurler's syndrome.
Nov 6, 2013
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