Congenital muscular dystrophy due to lamin A/C mutation

Clinical trials for Congenital muscular dystrophy due to lamin A/C mutation explained in plain language.

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

Disease control Expanded access (ended)

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…

Matched conditions: CONGENITAL MUSCULAR DYSTROPHY DUE TO LAMIN A/C MUTATION

Sponsor: Hope Biosciences Research Foundation • Aim: Disease control

Last updated Jun 27, 2026 07:52 UTC

Congenital muscular dystrophy due to lamin A/C mutation

Get alerted when new Congenital muscular dystrophy due to lamin A/C mutation trials appear

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