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Regulatory news: Olipudase alfa-rpcp (Xenpozyme™) for treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients-FDA Approval summary.

Yuen Yi HonAnita Zaidinull nullKathleen DonohueChristine Nguyen
Published in: Journal of inherited metabolic disease (2024)
Keyphrases
  • replacement therapy
  • transcription factor
  • young adults
  • drug administration
  • childhood cancer