Publication date: Jan 07, 2026
The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington’s disease (HD) compared to the control arm. HTT227 – current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.
| Concepts | Keywords |
|---|---|
| Blind | HD |
| Childbearing | HTT227 |
| Salpingectomy | HUNTINGTON DISEASE |
| Viral | INVEST-HD |
| Weeks | mHTT |
| NfL | |
| UHDRS | |
| VOTOPLAM |
Semantics
| Type | Source | Name |
|---|---|---|
| disease | MESH | Huntington’s Disease |
| disease | MESH | disease progression |
| disease | MESH | PTC |
| drug | DRUGBANK | Adenine |
| drug | DRUGBANK | Guanine |
| drug | DRUGBANK | Tilmicosin |
| disease | MESH | CAP |
| drug | DRUGBANK | Hepatitis B Vaccine (Recombinant) |
| disease | MESH | cardiac arrhythmias |
| disease | MESH | ventricular tachycardia |
| disease | MESH | AV block |
| disease | MESH | long QT syndrome |
| disease | MESH | Torsade de |
| pathway | KEGG | Huntington disease |
Original Article
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