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enzyme-replacement therapy

Coverage of enzyme-replacement therapy in the Nexus archive.

Earliest in view: Jun 22 · 10:43 UTCMost recent: Jun 22 · 10:43 UTC
Co-mentioned in this coverage
Regenxbioorganization1Food and Drug Administrationorganization1mucopolysaccharidosis type IItopic1Hunter syndrometopic1UniQureorganization1Huntington’s diseasetopic1Marty Makaryperson1
Recent coverage
  • HEALTHJun 22 · 10:43 UTCSTAT NEWS
    STAT+: FDA reverses course on Regenxbio’s childhood gene therapy after rejection

    The FDA will reconsider approving Regenxbio’s gene therapy for a rare childhood brain disorder, mucopolysaccharidosis type II (MPS II), after initially rejecting it four months ago. This follows recent FDA reversals, including allowing UniQure to resubmit a gene therapy application for Huntington’s disease previously rejected by former commissioner Marty Makary.