Huntington's Disease (HD)
Patient Condition
At the age of 35, the patient developed involuntary dance-like movements and emotional instability, which became progressive. Genetic testing confirmed that the HTT gene CAG repeats 48 times (causing disease>36), and Huntington's disease was diagnosed. At the time of diagnosis, the UHDRS total motor score was 42 points, and TMS was 30 points. Cognitive decline (MoCA 22 points) and mild depression had occurred.
Treatment Timeline
| Phase | Details | Date |
|---|---|---|
| WVE-003 ASO gene therapy evaluation | ||
| Combined comprehensive management of gene therapy | ||
| Long-term follow-up and functional maintenance |
Treatment Outcome
| before | UHDRS exercise score 42, MoCA 22, depression score 12, obvious dance-like movements |
|---|---|
| after | UHDRS exercise score 33 points (improvement 9 points), MoCA 25 points, depression relieved, return to work |
| highlight | Allele-selective ASO reduced mHTT by 35%, significantly improved exercise scores, and returned to normal work |