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  • ML4 DISEASE INFORMATION
    • Genetics of ML4
    • Diagnosis and Treatment
    • Clinical Care Suggestions
    • Glossary
  • RESEARCH
    • Introduction to Research
    • Our Priorities
    • Scientific Advisory Board
    • Tissue Donation: A Legacy of Love
  • SUPPORT FOR FAMILIES
    • Keep in Touch
    • Clinical Care Suggestions
    • Partner Organizations
    • Family Meetings
      • Family Meeting 6/13/2021
      • Dr. Misko 9/6/2020
      • 2020 Virtual Conference for Families
    • Our Family Stories
  • ABOUT THE ML4 FOUNDATION
    • History of ML4 Foundation
    • Message from the Executive Director
    • Board of Directors
    • Get Involved
      • Donate to The ML4 Foundation
      • Stretch Your Dollars for ML4
    • Contact Us

ML4 Glossary

Glossary of Useful Terms for Understanding Mucolipidosis Type IV:

  • AAV9
  • Agonist
  • Autophagy
  • Biomarker
  • Clinical Trial
  • CNS
  • CoRDS
  • CRISPR
  • Dendrites
  • Electromyography (EMG)
  • Endpoint
  • FMRI
  • Gene therapy
  • Genotype
  • Glial cells
  • Hematopoietic stem cells
  • Hypomylenation
  • Institutional Review Board (IRB)
  • Investigational New Drug Application (IND)
  • Leukodystophy
  • Lumbar Puncture
  • Lysosomal storage disease
  • Lysosome
  • Magnetic Resonance Imaging (MRI)
  • Microglia
  • Mucolipin-1 or MCOLN1
  • Mutation
  • Natural History Study
  • Neurodegenerative Disease
  • Neuroinflammation
  • Oligodendrocytes
  • Optical Coherence Tomography (OCT)
  • Pathogenesis
  • Pathway
  • Phenotype
  • PI
  • Prospective Natural History Study
  • Purkinjie cell
  • Registry
  • Retrospective Natural History Study
  • Small molecule
  • Stem cells
  • Substrate reduction therapy
  • Target
  • TFEB
  • Transduction
  • Transfection
  • Translational
  • Transmembrane protein defect
  • TRPML1
  • Tx
  • Vector
  • Wild type
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