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Ultragenyx's Gene Therapy for Sanfilippo Syndrome Receives FDA Acceptance
The FDA has accepted Ultragenyx's Biologics License Application for its gene therapy UX111, aimed at treating Sanfilippo Syndrome.
Editorial Staff
1 min read
Updated 1 day ago
Summary
Ultragenyx Pharmaceutical has announced that the FDA has accepted its Biologics License Application (BLA) for the gene therapy UX111, which is designed to treat Sanfilippo Syndrome.
This acceptance marks a significant step forward for Ultragenyx as it seeks to bring this treatment to patients affected by this rare genetic disorder.
The company is now poised to advance its efforts in addressing the needs of individuals with Sanfilippo Syndrome, pending further regulatory developments.
Key Facts
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Updates
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