Disease Library/Pitt-Hopkins syndrome/Advocacy Hub

Pitt-Hopkins syndrome

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At a Glance

Live from database
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1Recruiting trials
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1Specialists

Regulatory Watchboard

2 events
Dec 2025Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome

Mahzi Therapeutics — PHASE1, PHASE2

TrialRECRUITING
Oct 2025An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome

Unravel Biosciences, Inc. — PHASE1

TrialNOT YET 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 Pitt-Hopkins syndrome.
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Clinical Trial Landscape

1 active trial
1PHASE1, PHASE2
1Total recruiting
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Recent News & Research

No recent news articles indexed yet for Pitt-Hopkins syndrome.
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Specialist Network

Top 1 by expertise
JS

James Shaw

Neuren Pharmaceuticals

Score 133 trials20 pubs

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