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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One Child's hope: personal stem cells tested against rare muscle disease

Disease control NO_LONGER_AVAILABLE

This was a special access program for a single two-year-old child with a severe, rare genetic muscle disease. Doctors gave the child 14 infusions of his own specially processed stem cells over nearly a year to see if it was safe and if it could help control the disease. The study…

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

Sponsor: Hope Biosciences Research Foundation • Aim: Disease control

Last updated Apr 01, 2026 14:42 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

One Child's hope: personal stem cells tested against rare muscle disease