U.S., Nov. 4 -- ClinicalTrials.gov registry received information related to the study (NCT07223632) titled 'Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient' on Oct. 27.
Brief Summary: This is an 'N of 1', open-label, single center study to evaluate the safety of therapy with VCA-894A, an ASO designed to rescue and restore the activity of IGHMBP2, when administered by intrathecal injection.
Study Start Date: May 13
Study Type: INTERVENTIONAL
Condition:
Charcot Marie Tooth Disease (CMT)
Neuromuscular Diseases (NMD)
Intervention:
DRUG: VCA-894A
intrathecal antisense oligonucleotide injection
Recruitment Status: ACTIVE_NOT_RECRUITING
Sponsor: Vanda Pharmaceuticals
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