Gottlieb’s Aggressive Plans to Streamline the Orphan Drug Review Process

Thanks to Rare Disease Report for this article. On…

Rare Disease Report: Learning to See Thanks to a Blind Person
Many rare disease parents gain strength and inspiration by watching their ‘disabled’ child adapt because of – and sometimes thrive in… Continue Reading
Young Patient Collects Can Tabs to Raise Funds for Ronald McDonald House
(SOURCE) A Bristol teen who delivered a trailer full of aluminum can tabs to the Ronald McDonald House in 2014 is now attempting to collect… 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
Baby with Dextro-Transposition Finds Life-Long Success After Surgery
by  Natalie McGee-Lester I was born with dextro-transposition of the great arteries in Houston, Texas in 1975. During that time, d-TGA and… Continue Reading
RARECast: Delivering Enzyme Replacement Therapies Across the Blood-Brain Barrier
Enzyme replacement therapies are available for a number rare, metabolic diseases that collectively are known as lysosomal storage disorders.… Continue Reading
So Much More Than a Migraine: Idiopathic Intracranial Hypertension
by Amanda Ames Everyone in my family wears glasses. I remember being in first grade and struggling to see the chalkboard and being taken to… Continue Reading
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