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Neurologic, endocrine, and pancreatic disease, multisystem, infantile-onset

MONDO:0024189

1 clinical trial for this condition and its sub-types.

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Sub-types

Neurologic, endocrine, and pancreatic disease, multisystem, infantile-onset 2 (1) Neurologic, endocrine, and pancreatic disease, multisystem, infantile-onset 1 (0)

Broader categories

Disease (679) Hereditary disease (176) Human disease (14) Disease of genetic or genomic mechanism (2) Disease by etiologic mechanism (0)

Not yet finished but already full! 1

Rare disease study pulled before starting: scientists sought clues in cell stress

Knowledge-focused Terminated

This study was planned to learn how certain rare genetic mutations disrupt protein production and cause severe diseases affecting the lungs, liver, brain, and digestion. Researchers would have used cells from patients to measure protein levels and stress responses. However, the s…

Phase: NA • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused

Last updated Jun 27, 2026 07:51 UTC