Infantile Neuronal Ceroid Lipofuscinosis
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CLINICAL_TRIALS trial_result · 2025-02-17Clinical Trial Update: A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease [Phase 2]
Phase 2 safety, tolerability, and efficacy study of BMN 190 (cerliponase alfa) in pediatric patients under 18 with CLN2 disease has complete…
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CLINICAL_TRIALS trial_result · 2015-01-21Clinical Trial Update: Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis [Phase 4]
This Phase 4 clinical trial investigated the use of Cystagon (cysteamine bitartrate) to treat infantile neuronal ceroid lipofuscinosis; resu…
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CLINICAL_TRIALS trial_result · 2015-01-15Clinical Trial Update: Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL) [Phase 1]
A Phase 1 study evaluating HuCNS-SC neural stem cells in infants with infantile or late-infantile neuronal ceroid lipofuscinosis has complet…