PROGRESS IN RESEARCH.HOPE FOR AMARLI. ❤️
Promising gene therapy results in Rett syndrome give families reason to hope.
**A few words. Feeding yourself. Taking steps with support.
For a family living with a rare genetic condition, these can be life-changing milestones. ❤️ **Promising results from a gene therapy trial for Rett syndrome are giving families like ours reason to hope.
In an update from Taysha Gene Therapies, all 12 participants in the early-stage trial gained or regained at least one developmental milestone by 12 months after treatment.
Reported improvements included meaningful speech, eating independently with utensils and walking with support. Different participants experienced different gains, but behind each one is a person and a family whose everyday life could change.
Amarli has a different genetic condition, caused by a change in her GRIK2 gene. This Rett syndrome treatment is not a treatment for her condition, and these results do not establish a cure.
What they offer is an encouraging glimpse of what gene therapy may make possible.
Our hope is that lessons from trials like this will help researchers develop treatments for other rare genetic conditions.
CureGRIN is also planning research roadmaps for individual GRI disorders, including GRIK2, because each gene needs its own approach.
For our family, that hope is deeply personal.
We want Amarli to have every opportunity to communicate, develop new skills and enjoy a fuller, more comfortable life.
This is why Amarli Army keeps raising awareness and supporting research into rare genetic disorders. There is more work ahead, but progress like this gives us another reason to keep going.
For Amarli. For every family waiting. Never Never Never give up. ❤️
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