Natural History Study

**What it means:** A natural history study systematically tracks a disease **without giving any treatment**. It's like creating a detailed map of a territory before you try to change it — you need to understand the baseline, how things progress over time, what symptoms appear when, and how the disease affects different patients. **Why it's critical for MHS:** You can't design a clinical trial without knowing what "normal" looks like for MHS. How quickly does cognitive function decline? What symptoms appear first? What measures reliably track disease progression? The Volare Study and Boston Children's assessments are building this map. It also identifies the most promising clinical outcome measures that future treatment trials can use to prove the drug is working.