Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)

  • Trial registry record
  • Trial registry update

The n-Lorem Foundation, a nonprofit, made nL-GARS1-001, an antisense drug designed for one participant whose CMT2D is caused by a GARS1 deletion. The study runs at UTHealth Houston and enrols by invitation only.

No results have been posted. The registry expects the study to finish in October 2027.

Source date
Source updated
Registry status
Enrolling by invitation
Results posted to ClinicalTrials.gov
No
Genes
GARS1
Subtypes
CMT2D
Approved