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Gene Therapy Advances

The ML4 Foundation is funding several projects in gene therapy. One of these projects involves a collaboration between Dr. Steven Walkley (Einstein) and Dr. Charles Venditti (NIH). Read here about exciting developments in a gene therapy project also led by Dr. Venditti emerging in NPC disease. The ML4 project is modeled closely on this NPC project.

NIH Gene Therapy Press Release

CoRDS patient registry a success!


Terrific enrollment by ML4 families in our CoRDS patient registry! In 2015, the ML4 Foundation launched our disease-specific patient registry with Sanford CoRDS. This registry contains clinical records by over 27 MLIV families who have taken the time and care to document how the disease manifests itself in their child. The questions, developed by MLIV scientists and with input from MLIV families, cover all aspects of the disease. Families will be asked to update this registry annually. Researchers can now access this de-identified data by contacting CoRDS directly.

Funded Research

The ML4 Foundation Board of Directors is pleased to report continued funding to:

  • Dr. Yulia Grishchuk (Massachusetts General Hospital) for her proposal, Targeting Neuroinflammation to Treat Mucolipidosis Type IV
  • Dr. Tony Futerman (Weizmann Institute) for his proposal, Gene Array Analysis and Gene Expression in MLIV
  • Dr. Diego Medina (TIGEM) for his proposal, High content imaging approaches to investigate TRPML1 modulation and identify compounds inducing clearance in Mucolipidosis type IV.

See more about our funded research at Funded Research. screens compounds for MLIV!

Read about the exciting progress at in their efforts to screen for compounds to improve MLIV disease. “From Dead to Alive” certainly got our attention!