• Brineura

  Form: solution for infusion

  Active substance	Dosage
  CERLIPONASE ALFA	—

  ATC: A16AB17

• Kuvan

  Form: tablets, soluble

  Active substance	Dosage
  Sapropterin dihydrochloride	—

  ATC: A16AX07

• Naglazyme

  Form: solution for infusion

  Active substance	Dosage
  GALSULFASE	—

  ATC: A16AB08

• Palynziq

  Form: solution for injection

  Active substance	Dosage
  PEGVALIASE	—

  ATC: A16AB19

• Roctavian

  Form: solution for infusion

  Active substance	Dosage
  VALOCTOCOGENE ROXAPARVOVEC	—

  ATC: B02

• Vimizim

  Form: solution for infusion, concentrate

  Active substance	Dosage
  ELOSULFASE ALFA	—

  ATC: A16AB12

• Voxzogo

  Form: solution for injection, powder and solvent

  Active substance	Dosage
  VOSORITIDE	—

  ATC: M05BX07