Research & Clinical Trials

Exploring Family Member Perceptions and Experiences with Genetic Testing for ALSP

Allo HCT for Metabolic Disorders and Severe Osteopetrosis

  • Interventional

  • Masonic Cancer Center, University of Minnesota

  • Insurance Pre-Approval

  • Dr. Troy Lund

  • Name: Troy Lund, MD, PhD

    Phone Number: 612-625-4185

    Email: lundx072@umn.edu

Precision Genome Editing for CSF1R-Related Disorders

  • Preclinical

  • University of California San Francisco

    Massachusetts General Hospital

  • Free to participate. Travel costs may be covered through a grant.

  • UCSF: Dr. Jeffrey Gelfand

    MGH: Dr. Yedda Li

  • Name: Yedda Li, MD

    Email: yli@mgh.harvard.edu

  • Observational: Natural History Study

  • Children’s Hospital of Philadelphia

  • Free to participate. Travel costs paid out of pocket.

  • Dr. Jennifer Orthmann-Murphy

  • Name: Omar S. Sherbini, MPH

    Phone Number: 215-590-3068

    Email: sherbinio@chop.edu

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

  • Observational

  • Assistance Publique - Hôpitaux de Paris, France

  • Contact for additional information.

  • Dr. Fanny Mochel

  • Name: Fanny Mochel, MD, PhD

    Phone Number: 01 57 27 44 82

    Email: fanny.mochel@icm-institut.org

Combining the science and humanity of microgliopathies.