Health ❯ Pediatrics

Rare Diseases

Genetic Disorders Urea Cycle Disorders Genetic Testing Cancer Treatment SMA Treatments Neuromuscular Disorders Treatment Options CPS1 Deficiency Innovative Therapies Metabolic Disorders Brain Tumors Wolf-Hirschhorn Syndrome Barth Syndrome Diagnosis

BrainIAC AI Trained on 49,000 MRIs Outperforms Rivals Across Dementia and Cancer Tasks

Self-supervised training on nearly 49,000 MRIs enabled broad, data-efficient gains, with clinical deployment contingent on further validation.

Nature Study Flags ABHD18 Inhibition as a Preclinical Path to Treating Barth Syndrome

Science Advances Study Identifies Pentose Phosphate Pathway Vulnerability in MPNST

Small-Molecule Precursors Restore CoQ10 and Reverse Neurological Decline in HPDL Deficiency

Proteomic Test Cuts Childhood Rare Disease Diagnosis to Under Three Days