A Mother's Fight: Saving her Daughter from a Rare Genetic Disease

TLDRMila, a once healthy and active child, was diagnosed with Batten Disease at the age of six. Her mother, Julia, founded M's Miracle Foundation to raise funds for research and potential treatments. Along the way, Julia discovered a missing mutation and found new hope in gene therapy. Despite the challenges, Julia remains determined to fight for her daughter's life.

Key insights

💪Mila's rapid decline in health due to Batten Disease

🌟Julia's founding of M's Miracle Foundation to fund research and potential treatments

🔬The search for a missing mutation and the discovery of new hope in gene therapy

😭Julia's emotional journey and determination to fight for her daughter's life

💡The potential impact of spinraza as a treatment for Batten Disease

Q&A

What is Batten Disease?

Batten Disease is a rare genetic disorder that causes a progressive decline in physical and mental abilities.

How did Julia respond to Mila's diagnosis?

Julia founded M's Miracle Foundation and dedicated her efforts to raising funds for research and potential treatments for Batten Disease.

What is gene therapy?

Gene therapy involves introducing genetic material into a person's cells to correct or replace faulty or missing genes.

Has gene therapy shown promise for Batten Disease?

Gene therapy holds potential as a treatment for Batten Disease, but further research and development are needed.

What is spinraza?

Spinraza is a drug approved for the treatment of spinal muscular atrophy, but it has also shown potential for other genetic diseases.

Timestamped Summary

00:00Introduction and background of the video

05:28Mila's diagnosis and Julia's response to it

10:57The search for a missing mutation and the discovery of gene therapy

16:04Julia's determination and the founding of M's Miracle Foundation

20:19The potential impact of spinraza as a treatment for Batten Disease

25:42Conclusion and call to action