Research · April 14, 2026
Research Identifies New Genetic Strategy to Treat Rett Syndrome
New research identifies potential treatment pathway for rare Rett Syndrome, offering hope where none existed before....
Treatment · March 31, 2026
FDA Grants Priority Review for First Targeted Therapy for Severe Genetic Epilepsies
FDA accepts priority review of new drug for rare developmental epilepsy, offering hope to affected families....
Treatment · March 25, 2026
FDA Approves First Brain-Penetrating Therapy for Hunter Syndrome in Decades
FDA approves the first new Hunter Syndrome treatment in nearly two decades, offering hope to patients with this rare genetic disea...
Breakthrough · March 23, 2026
FDA Grants Priority Review for First Potential Treatment for Fatal Neurological Disease Alexander Disease
FDA accepts zilganersen for Priority Review to treat Alexander disease, offering hope to patients with this rare condition....
Breakthrough · March 5, 2026
Scientists Identify Promising New Treatment Pathway for Rare Rett Syndrome
Researchers have identified a promising new treatment pathway that offers hope for Rett Syndrome, a rare disorder without current ...
Breakthrough · January 14, 2026
Breakthroughs Bring First Potential Treatments for Huntington's Disease Closer
Multiple research breakthroughs bring hope for Huntington's disease treatments on the horizon....
Breakthrough · December 15, 2025
Gene Therapy Breakthrough Shows Promise for Rare Neurological Disorder
Gene therapy breakthrough offers new hope for patients with hereditary spastic paraplegia....
Research · October 21, 2025
From Lab to Life, Gene Therapy Offers New Hope for Neurological Disorders
Translational research successes demonstrate how laboratory discoveries are transforming into real-world neurological treatments....