Research & Clinical Trials
Global Patient Registry
for Microgliopathies
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Observational
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N/A
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CoRDS and GliaConnect
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Email: erin@gliaconnect.org
Allo HCT for Metabolic Disorders and Severe Osteopetrosis
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Interventional
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Masonic Cancer Center, University of Minnesota
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Insurance Pre-Approval
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Dr. Troy Lund
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Precision Genome Editing for CSF1R-Related Disorders
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Preclinical
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Massachusetts General Hospital
University of California San Francisco
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Free to participate. Travel costs may be covered through a grant.
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MGH: Dr. Yedda Li
UCSF: Dr. Jeffrey Gelfand
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MGH
Name: Yedda Li, MD
Email: yli@mgh.harvard.edu
UCSF
Name: Dr. Jeffrey Gelfand
Email: jeffrey.gelfand@ucsf.edu
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Observational: Natural History Study
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Children’s Hospital of Philadelphia
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Free to participate. Travel costs paid out of pocket.
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Dr. Jennifer Orthmann-Murphy
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The Myelin Disorders Biorepository Project
Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
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Observational
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Assistance Publique - Hôpitaux de Paris, France
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Contact for additional information.
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Dr. Fanny Mochel
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