# Pharmacological chaperone treatment with Cystadane in aspartylglucosaminuria: an open-label, phase Ib/II trial

Source: [nature.com](https://www.nature.com/articles/s41467-026-78752-x) · 2026-11-11 · Life
Page: https://themorning.science/story/pharmacological-chaperone-treatment-with-cystadane-in-aspartylglucosaminuria-an-4f3481b5b8854f13
Kind: research · Impact: medium

## In brief

- An open-label phase Ib/II clinical trial tested Cystadane (betaine) as a pharmacological chaperone in aspartylglucosaminuria.
- The trial reports that betaine/Cystadane is safe to use.
- Treatment significantly reduced urinary accumulation of glycoasparagines, according to the published abstract.

## Why it matters

It provides early clinical evidence that an existing, orally used compound may be a candidate treatment for a rare inherited metabolic disorder, which matters for clinicians and researchers working on rare diseases.

## Timeline

- 2026-11-11: Trial results published online in Nature Communications

## Coverage

- [Pharmacological chaperone treatment with Cystadane in aspartylglucosaminuria: an open-label, phase Ib/II trial](https://www.nature.com/articles/s41467-026-78752-x) — nature.com, 2026-11-11

Tags: rare disease, clinical trial, pharmacological chaperone, betaine, aspartylglucosaminuria, metabolism

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Brief generated by AI (claude-haiku-5-5) from the reporting listed under Coverage. Headlines belong to their publishers. The Morning Science – https://themorning.science