Context

Labrune Syndrome/LCC ("Leukoencephalopathy, Cerebral Calcifications, and Cysts") is an ultra-rare, progressive neurological disease with no FDA-approved treatment and limited awareness in the medical community.

First, LCC is a devastating disease that causes brain calcifications, cysts, and progressive neurological decline. Very little research exists, which means that doctors have limited knowledge and there are no standardized treatment protocols. Without funding, clinical trials will not happen, leaving families without hope for therapeutic options.

Moreover, families affected by LCC often struggle to find knowledgeable doctors, obtain an accurate diagnosis, and access appropriate medical care. Many caregivers feel alone, without a support system or guidance to manage their child’s symptoms. The financial burden is overwhelming, whether for medical expenses, specialized equipment or travel for care.

We need to accelerate research to find effective treatments and, ultimately, a cure, and provide resources and contacts to ease these challenges and empower families.

Goals

To create a future where those diagnosed with LCC/Labrune Syndrome have hope, better care, and, one day, a cure.

Activities

• Research funding and support: providing financial support and collaborating with scientists to drive research into LCC/Labrune Syndrome, focusing on understanding disease mechanisms, potential drug targets, and therapeutic development;
• Family Support: offering affected families direct assistance through information and connections to specialists.

Myriad Canada is working with The LCC Foundation on this project, a nonprofit whose mission is to support families affected by LCC across the United States and globally, and to provide resources to help fight this rare and debilitating disease. The Foundation focuses on funding research projects aimed at discovering novel treatments, therapies, and ultimately a cure for LCC. It also strives to raise awareness about LCC, to improve the chances of early diagnosis and intervention.