A Global Alliance to Save Lives: How Two Parents and PackGene Are Redefining What’s Possible in Gene Therapy with Help from Weill Cornell Medicine

HOUSTON, Jan. 8, 2025 /PRNewswire/ — Two Australian parents, faced with the unimaginable prospect of losing their 14-month-old daughter to the ultra-rare neurological disease Hereditary Spastic Paraplegia Type 56 (SPG56), sold their home and everything they owned to save their daughter….