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A total of 917 posts are filed under Insights
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
My Wife Had Ehlers Danlos Syndrome and She Never Knew
My wife died in 2008. It was the same year my daughter was diagnosed with Ehlers Danlos Syndrome (EDS). We have dedicated a new program in… Continue Reading
WIRED: A Radical Proposal For Preventing Rare Genetic Diseases
(SOURCE) One morning, A few months ago, I didn’t wake up. My 29-year-old heart had suffered from a sudden and complete cardiac arrest as I… Continue Reading
Kyle Bryant Continues Athletic Pursuits Long After Diagnosis of Friedreich’s Ataxia
SOURCE — Kyle Bryant was a kid who wanted the ball. He wanted in on the game and always wanted a chance to help his team win. Growing up… Continue Reading
RARECast: Sangamo Advances Gene Therapies for Multiple Rare Diseases Into Clinical Development
The promise of gene therapy has rare disease patients not only contemplating the potential of new treatments, but ones that can free them… Continue Reading

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