Active, not recruitingPHASE1, PHASE2NCT06816498

Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

Studying Adult-onset autosomal dominant leukodystrophy

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Key facts

Sponsor
n-Lorem Foundation
Intervention
nL-LMNB1-001(drug)
Enrollment
1 enrolled
Eligibility
51-51 years · MALE
Timeline
20252027

Study locations (1)

Collaborators

Mayo Clinic

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

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