Phelan-McDermid syndrome

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At a Glance

Live from database

Regulatory Watchboard

4 events
Nov 2025A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome

Neuren Pharmaceuticals Limited — PHASE3

TrialRECRUITING
Jun 2025RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)

Peking University First Hospital — NA

TrialRECRUITING
Jan 2024JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency

Jaguar Gene Therapy, LLC — PHASE1, PHASE2

TrialRECRUITING
May 2015Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome

Boston Children's Hospital

TrialACTIVE NOT RECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

0

Total programs

No financial assistance programs currently listed for Phelan-McDermid syndrome.
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Clinical Trial Landscape

4 active trials
1Phase 3
1N/A
1Unknown
1PHASE1, PHASE2
4Total recruiting
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Recent News & Research

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Specialist Network

Top 6 by expertise

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