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Gene Therapy Advances

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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

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CoRDS patient registry a success!

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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.

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Newly Funded Research

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The ML4 Foundation Board of Directors is pleased to report approval of funding for Dr. Christian Grimm (University of Munich) for his proposal, Known and novel members of the endolysosomal transportome/channelome as candidates to rescue loss of TRPML1 function in MLIV patients, and to Dr. Diego Medina (TIGEM) for his proposal, High content imaging approaches to investigate TRPML1 modulation and identify compounds inducing clearance in Mucolipidosis type IV.

We are also eagerly anticipating results from recently funded research by Dr. Kirill Kiselyov (University of Pittsburgh), GDAP1 inhibitors as a treatment option for Mucolipidosis Type IV

Each of these projects focusses on treatment options for ML4 patients.

Sarah Zar Purim

Exciting New Investigations at biotech startup, Perlara PBC!

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The ML4 Foundation is pleased to announce that Perlara PBC, a Bay Area biotech created to discover personalized cures for diseases rare and common will begin research on Mucolipidosis Type IV. Perlara (www.perlara.com) “creat[es] disease models using simple animals that share genetic similarity with humans, allowing [them] to screen massive numbers of disease models and drug candidates quickly and at low cost.” Perlara has begun utilizing their unique platforms in conjunction with models shared from the generous MLIV scientific community to search for potential drugs for this disease. Stay tuned for official announcement soon!