On Sunday, August 19, Taylor King will turn 20. On November 1, Laura King Edwards’s inspiring book “Run To The Light” will be published, by Bedazzled Ink. The memoir is an astonishing tale ofCategory Archives: rare disease
New Book, “Run to the Light,” Chronicles a Journey with Batten Disease
On Sunday, August 19, Taylor King will turn 20. On November 1, Laura King Edwards’s inspiring book “Run To The Light” will be published, by Bedazzled Ink. The memoir is an astonishing tale ofNo Ice Buckets or Pink Ribbons for Very Rare Genetic Diseases
As enthusiasm for dumping ice on one another fades with autumn and October brings pervasive pink, I wish that attention would turn to families confronting diseases not as well known as ALS and breast cancer. HOW RARE IS RARE? According … Continue reading
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Wilson Disease – A Genetic Success Story
A woman I never met, Ingrid, has taught thousands of students about Wilson disease, an inborn error of copper metabolism. But she never knew it, and I wish I could tell her.
COPPER CONTROL GONE AWRY
Wilson disease is …
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Eliza’s Journey: Part 2
Gene therapy is, finally, about to take off! The feeling was palpable at the American Society of Gene and Cell Therapy annual meeting a few weeks ago. It’s been nearly a quarter century since the first experiment in humans.
The drug …
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Posted by in ASGCT, gene therapy, Lysogene, NORD, rare disease, Sanfilippo syndrome
Of Tissue-Engineered Vaginas and Default Options
I’m old enough to remember textbooks and biology classes that defined being female as a “default option” in human development.
If the ambiguous sex organ precursors in an embryo “failed” to follow the coveted male route, we became female.
If …
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