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Regulatory news: Cipaglucosidase alfa-atga (Pombiliti) coadministered with Miglustat (Opfolda) for adults with late-onset Pompe disease.

Daniela V LuquettiLinda J B JengKathleen M DonohueJanet W Maynardnull null
Published in: Journal of inherited metabolic disease (2024)
Keyphrases
  • late onset
  • early onset
  • transcription factor
  • replacement therapy
  • smoking cessation