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Children's Hospital of Philadelphia (1)

Biogen (1)

Foundation to Fight H-ABC (1)

Homology Medicines, Inc (1)

National Institute of Neurological Disorders and Stroke (NINDS) (1)

National Institutes of Health (NIH) (1)

PMD Foundation (1)

Pennsylvania Department of Health (1)

Takeda Pharmaceutical Co. Limited (1)

Other (1)

Observational [Patient Registry] (1)

peroxisomal biogenesis disorder

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Dec 21, 2021