Pelizaeus-Merzbacher disease Advocacy Hub | UniteRare

At a Glance

Live from database

Regulatory Watchboard

5 events

Jul 2026A Phase 2 Study of Radiotherapy-induced Immune Priming to Enhance Elranatamab (Elra) in Relapsed Refractory Multiple Myeloma (RRMM) With Extramedullary Disease (EMD) and Paramedullary Disease (PMD) "PRIME-EMD-PMD"

M.D. Anderson Cancer Center — PHASE2

TrialNOT YET RECRUITING

Jan 2025Mesh-Free Versus Mesh-Based Surgery for Female Stress Urinary Incontinence: A Prospective Comparison of Pubo-Urethral Ligament Plication and Transobturator Tape

Mehmet Incebıyik — NA

TrialRECRUITING

May 2024Collection of Blood and Skin Samples From Patients With Primary Mitochondrial Diseases and Healthy Volunteers

Minovia Therapeutics Ltd.

TrialRECRUITING

Apr 2024Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

Ionis Pharmaceuticals, Inc. — PHASE1

TrialRECRUITING

Oct 2022Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

Ionis Pharmaceuticals, Inc.

TrialRECRUITING

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

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

Total programs

No financial assistance programs currently listed for Pelizaeus-Merzbacher disease.
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Clinical Trial Landscape

2 active trials

1Phase 1

1Unknown

2Total recruiting

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Recent News & Research

No recent news articles indexed yet for Pelizaeus-Merzbacher disease.
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Specialist Network

Top 6 by expertise

Adeline Vanderver, MD

Children's Hospital of Philadelphia

📍 Los Angeles, California

Score 154 trials1 pub

Joanne Kurtzberg, MD, M.D

Duke University

📍 DURHAM, NC

Score 1111 trials

Mİ

MEHMET İNCEBIYIK, MD

Harran University

Score 31 trial

Stephen Huhn, MD, MD

StemCells, Inc.

📍 STANFORD, CA

Score 34 trials

Gail Hartin

Ionis Pharmaceuticals

Score 01 pub

Jordan McSherry, lpn

Ionis Pharmaceuticals

📍 YAPHANK, NY

Score 01 pub

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