Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

Key facts

Sponsor

Massachusetts General Hospital

Principal Investigator

Florian Eichler, MD

Massachusetts General Hospital

Intervention

Antisense oligonucleotide treatment (ASO)(drug)

Enrollment

1 target

Eligibility

4 years · All sexes

Timeline

2024 – 2026

Study locations (1)

• Massachusetts General Hospital, Boston, Massachusetts, United States

Collaborators

n-Lorem Foundation

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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