For Patients & Caregivers

Disease Library

In-depth guides on genetic diseases — biology, daily management, treatment options, clinical trials, and community resources.

Featured guides

Comprehensive disease guides with treatment landscapes, clinical trials, and community resources.

An inherited disorder that causes severe damage to the lungs, digestive system, and other organs.

A progressive genetic disorder causing muscle degeneration and weakness, primarily affecting boys.

A rare metabolic disorder causing extreme photosensitivity and painful skin reactions to sunlight.

A group of inherited blood disorders affecting hemoglobin, causing pain crises, organ damage, and reduced life expectancy.

A genetic neuromuscular disease causing motor neurons to deteriorate, leading to progressive muscle weakness.