Gene Therapy Success in Rare Diseases

This Mom’s Epic Push to Save Her Son’s Life Will Leave You in Awe

Celebrating The Moms of Gene Therapy | DNA Science Blog

In 18 months we moved from the first-ever meeting of scientific investigators addressing an expedited path toward treatment or cure for CMT4J, to proof-of-concept in our preclinical work, raising over $1 million towards a gene therapy.

Gene therapy?s new hope: A neuron-targeting virus is saving infant lives
The gene-delivering virus may give scientists a new way to treat devastating spinal and brain diseases

Sickle cell anemia patient 'cured' by gene therapy, doctors say
In a world first, a teenager with sickle cell disease achieved complete remission after an experimental gene therapy.
Hemophilia cure? Gene therapy trial shows dramatic results
A year after gene therapy treatment, people with hemophilia A are showing normal levels of clotting factor and big reductions in bleeding.
Two Months After Gene Therapy, Jack Sees Brighter, More Clearly - FOCUS - A health blog from Mass. Eye and Ear
Jack was born with an eye condition known as retinitis pigmentosa associated with RPE65 gene mutations. On March 20, 2018, he was treated with a newly FDA-approved gene therapy drug known as Luxturna and the results are life-changing.

 

When the O?Neills learned that their daughter had Sanfilippo syndrome, a devastating rare disease, they created a GoFundMe campaign that raised $2 million in less than a year.
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