What is the new drug for Niemann-Pick disease?
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3 min read
For decades, there were no U.S. Food and Drug Administration (FDA)-approved treatments available to address the underlying causes of Niemann-Pick disease. This has recently changed with the approval of several new medications, fundamentally altering the therapeutic landscape for this rare genetic disorder. The question, 'What is the new drug for Niemann-Pick disease?' now has a multifaceted answer, with different treatments available for specific types of the condition.
