Latest gene therapy trials herald good news as drug developments escalate

It’s much too soon to tell whether the gene therapy that 12-year-old Hannah Sames had last month for GAN is having an effect…

[Hannah’s mother] hopes Hannah’s increasing strength reflects motor neurons producing the gigaxonin protein that her two deleted genes make impossible…Perhaps Hannah’s disease will slow or stop, and she may even regain some feeling in her legs.

Eliza O’Neill is only about 2 months ahead of Hannah, but already biochemical signs and perhaps observations suggest that her gene therapy is working. Eliza has Sanfilippo syndrome type A.

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Eliza’s parents are noticing a difference…since the gene therapy. They try to be objective, but that’s hard when they’ve watched their little girl fade away, losing all ability to communicate.

“Since treatment, we see a new light in Eliza’s eyes— she is connecting with us in a way we thought was gone forever. She is working to regain the skills the disease stripped away in the 6 and half years it tore through her body. She is a fighter!” said her father Glenn.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion and analysis. Read full, original post: 3 Gene Therapy Trials Report Good News

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