How two sisters mastered the technology of critical care to help their mother live with ALS. Darji was a profoundly devout man. His piety...

This is the story of how a family dodged the severely debilitating rare disease - Spinal Muscular Atrophy

A new cellular model of GNE Myopathy reveals disease mechanism and holds promise for testing potential therapies. Jason Doles & Team at...

The world doesn’t care when a disease is rare, while a parent doesn’t care, that the disease is rare . Over the ages, it’s parents who...