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Browse Studies — Arctuva | Arctuva

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A Prospective Observational Study to Assess the Long-term Safety and Immunogenicity of Olipudase Alfa Therapy During Routine Clinical Care in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency

US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (…

Niemann-Pick DiseasesAcid Sphingomyelinase Deficiency

~$10 – $1,125

SanofiNCT06192576

Longitudinal Study of Neurodegenerative Disorders

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understandi…

MLDKrabbe DiseaseALD+24 more

~$10 – $6,250

University of PittsburghNCT03333200