Desperate hope: stem cells tested on one child with rare muscle disease

NCT ID NCT05154851

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This trial gives a single child with a rare form of congenital muscular dystrophy access to their own banked stem cells. The cells are given through 14 IV infusions to see if they are safe and can help control the disease. Because it involves only one patient, the results will be very limited.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

autologous adipose-derived mesenchymal stem cells (HB-adMSCs)

What this could lead to

If successful, this could point toward a way to slow or manage congenital muscular dystrophy in this patient.

What could go wrong

This is a single-patient expanded access study, not a full clinical trial. Results may not apply to others, and risks include infection or infusion reactions.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

congenital muscular dystrophy due to LMNA mutation muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Hope Biosciences Stem Cell Research Foundation

    Sugar Land, Texas, 77478, United States