Clinical Trial Update: RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS) [NA]

View source ↗

Summary

RB001 gene therapy is being studied in pediatric patients with SHANK3-related Phelan-McDermid Syndrome and the trial is currently recruiting.

Upgrade for the full analysis, importance score, and drug timeline.

Diseases / indications

• Phelan-mcdermid Syndrome
• Shank3 Haploinsufficiency

Drugs mentioned

• RB001

Snippet

Sponsor: Peking University First Hospital | Conditions: Phelan-McDermid Syndrome, SHANK3 Haploinsufficiency | NA | Status: RECRUITING