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Neuronal Ceroid Lipofuscinosis 2 - 3 Studies Found

Status Study
Completed Study Name: A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease
Condition:
  • Jansky-Bielschowsky Disease
  • Batten Disease
  • Jansky-Bielschowsky Disease
  • Batten Disease
  • Jansky-Bielschowsky Disease
  • Batten Disease
  • Drug: BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1)