Cure Mucolipidosis

Cure Mucolipidosis (Cure ML) is an international patient-driven organisation dedicated to bringing hope, treatment, and ultimately a cure to individuals and families affected by the ultra-rare Mucolipidosis disorders such as Mucolipidosis alpha/beta, Mucolipidosis Gamma and Sialidosis. For decades, ML has been a forgotten group of diseases with no approved therapies. Cure ML exists to change that.
We work to accelerate the development of life-changing treatments—especially gene therapy, which has shown real promise for this community after years of scientific stagnation. Through our strategic partnership with UMass Chan Medical School, Cure ML works with the team to support the full research pathway: from early discovery through preclinical development and into clinical trials.
Our mission goes beyond science. We stand beside families every step of the way—providing information, connecting communities worldwide, advocating for patient needs, and raising the significant funding required to push rare-disease research forward. Our work is built on compassion, collaboration, and a relentless determination to ensure no family faces ML alone.
Cure ML is powered by families and supporters, and driven by the belief that together, we can turn hope into reality.