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Story perspectives

Montana Family Seeks Hope in Custom ALS Treatments

9/2/2025

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Story summary
  • A family in Montana is seeking assistance from St. Jude Children's Research Hospital for their daughters with a rare genetic mutation causing severe seizures.
  • Researchers are investigating personalized treatments for ALS and other rare genetic diseases, yielding promising results with custom-built drugs.
  • Early cases demonstrate significant improvements, prompting a global trial for "N-of-1" medicine.