Eleven-year-old Hannah Sames can still curl her toes, just barely. But time is running out. If Hannah can move her toes for a few more weeks, until she becomes the fourth child in a clinicalCategory Archives: giant axonal neuropathy
Hannah’s 2016: From Curling Toes to Gene Therapy
Eleven-year-old Hannah Sames can still curl her toes, just barely. But time is running out. If Hannah can move her toes for a few more weeks, until she becomes the fourth child in a clinicalPosted by in clinical trials, featured, GAN, gene therapy, giant axonal neuropathy, Hannah Sames, Lori Sames, NORD, physical therapy, Steven Gray, Xenokeryx
Midsummer Updates at DNA Science
Summer is half over, so I thought I’d update a few posts. EMAN IN LIBERIA A year ago, I frantically wrote about my young friend in Liberia, Emmanuel Gokpolu, and his pleas to help stop Ebola. Emmanuel and his loved ones … Continue reading
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Hannah’s Hair – Why Traits Matter
It’s an unacknowledged law of nature that whatever the texture of a girl’s hair, she wants the opposite. For years I wrapped my tangles around soup cans and around my head, squished it under irons, and subjected it to stinky … Continue reading
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AGTC Tackles 3 Eye Diseases with Gene Therapy
September will be 25 years since the first gene therapy experiment, and FDA approval is finally in sight. Several gene therapies are approaching the finish line, awaiting results from comparisons to existing therapies and analyses of long-term efficacy. Among the … Continue reading
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No 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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Catching Up With 3 Rare Disease Families
Four-year-old Eliza O’Neill’s viral videos, the subject of my last two blog posts, continue to dominate the news media with another appearance on The Today Show June 17. Hopefully, her family’s fight to fund gene therapy for her …
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Eliza’s Journey: Part 1
If ten science writers were asked to write a book about gene therapy, a biotechnology with roots going back to the 1950s, they could tell ten different stories.
VARIATIONS ON THE THEME
Any account of gene therapy would include the …
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