As the first executive director of the ML4 Foundation, I work with patients, families, investigators, government organizations, industry, clinicians, caregivers, and other stakeholders to 1) create a better understanding of Mucolipidosis Type IV as a disease and as a community of people and 2) develop a strategy for creating a treatment for this complex genetic disorder.
I enjoy wearing many hats in this position. I am fortunate to speak with many families regularly and I use these conversations to understand your needs and communicate them to the investigators working on treatments. I am honored to seek out and collaborate with many scientists who are eager to understand the MLIV community and the unmet need among the patients. I am grateful for the many partnerships we have forged among engaged donors who are interested in making profound change in the world through creating gene therapy for MLIV disease. And I am delighted in bringing all of these forces together on behalf of really wonderful children. The ML4 Foundation is the only patient advocacy group in the world working on this disease, and we serve families in the United States, Canada, South America, the UK, France, South Africa, Australia, Israel, Turkey, Japan, and other locations.
