Patient Story

Rare Patient Starts Card Project for Hospitalized Kids
SOURCE Jen Rubino knows how it feels to be sick and in the hospital at a young age. The 22-year-old was diagnosed with a rare childhood bone… Continue Reading
Perth Family Confronts Metachromatic Leukodystrophy Diagnosis in Newborn
by Liam Croy A Perth family devastated by a rare genetic disorder has been thrown a lifeline by a world-leading research program in Italy.… Continue Reading
Tunnel Vision, Headaches Lead to Rare Diagnosis of Paroxysmal Nocturnal Hemoglobinuria
by Skyler Hrabe I will never forget the feeling of utter helplessness, despair and disbelief as the realization dawned on me that my… Continue Reading
First Duchenne Muscular Dystrophy Boy in Canada to Begin Drisapersen Redosing
Five years ago, 12-year-old Alex Chiabai was one of the first boy in Canada to begin taking drisapersen, a promising drug that provided a… Continue Reading
Australian Man Experiences Rare Instance of Guillain-Barré Syndrome (GBS)
Thanks to 9News.com.au for this piece. A Sydney man who was paralysed by a rare autoimmune disease while holidaying in New Zealand last… Continue Reading
Brother and Sister Receive Rare Cystinosis Diagnosis
Joel Strickland is a rare boy, not only because he is a diminutive, 12-year-old, guitar-playing, cowboy-hat-wearing, dirt-bike-riding… Continue Reading
British Woman Diagnosed with Ischemic Oesophagus, First Mistaken as a Heart Attack
A widow has become the first Briton ever to be diagnosed with an ultra-rare disease after she regurgitated seven pints of vomit that looked… Continue Reading
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