Latest Advances

  • Condition: Neuronal Ceroid Lipofuscinosis Type 2
  • Journal: Journal of child neurology
  • Treatment Used: Cerliponase Alfa
  • Number of Patients: 0
  • Published —
In this study, researchers evaluated the safety and effectiveness of cerliponase alfa for the treatment of neuronal ceroid lipofuscinosis type 2.
  • Condition: CLN2 Disease
  • Journal: Clinical immunology (Orlando, Fla.)
  • Treatment Used: Intracerebroventricular (ICV)-Delivered Cerliponase Alfa Enzyme Replacement Therapy (ERT)
  • Number of Patients: 24
  • Published —
This study evaluated the safety and effectiveness of intracerebroventricular (ICV)-delivered cerliponase alfa enzyme replacement therapy (ERT) (injection directly into the cerebrospinal fluid to bypass the blood-brain barrier) for the treatment of patients with CLN2 disease.
  • Condition: Ceroid Lipofuscinosis Type 2 Disease
  • Journal: The New England journal of medicine
  • Treatment Used: Intraventricular Cerliponase Alfa
  • Number of Patients: 24
  • Published —
The study researched the use of intraventricular cerliponase alfa for treating ceroid lipofuscinosis type 2 disease.
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