Tag Archives: rare diseases

A world of hope: Treating international patients with rare diseases at Children’s Hospital Oakland

Meet Nomin Gambat, a 5-year-old girl who traveled all the way from Mongolia to Children’s Hospital Oakland to seek a clinical treatment for a disease so rare it strikes only 1 in a million people. But coming...
Faces of Morquio patients

Taking a shot: An orphan drug trial that drew an international crowd of patients nears its end

When Sarah Morrell was first diagnosed with Morquio syndrome, a rare, incurable disease that crippled her joints and deformed her vertebrae, her family never expected the illness would cause her to travel halfway around the...