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Clinical trial

The Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient With Confirmed IGHMBP2 Intronic Cryptic Splice Variant C. 1235+894C>A That is Amenable to Antisense Oligonucleotide (ASO)-Mediated Correction of IGHMBP2 Splicing

NCT07223632 is registered in ClinicalTrials.gov with Vanda Pharmaceuticals as lead sponsor.

NCT NCT07223632
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Registry record

As filed in ClinicalTrials.gov.

Title
The Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient With Confirmed IGHMBP2 Intronic Cryptic Splice Variant C. 1235+894C>A That is Amenable to Antisense Oligonucleotide (ASO)-Mediated Correction of IGHMBP2 Splicing
Lead sponsor
Vanda Pharmaceuticals
Phase
Phase 1/2
Overall status
Active, not recruiting
Start date
May 13, 2025
Completion date
March 31, 2027
Enrollment
1 (actual)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

charcot marie tooth disease (cmt), neuromuscular diseases (nmd)