Generative AI: Transforming Hope for Rare Disease Patients Millions of people worldwide face the daily reality of rare diseases, many of which have no approved therapies. Traditional drug development often overlooks these conditions due to high costs and limi... AI drug discovery ALS biotechnology clinical trials generative AI IPF medical research rare diseases
Personalized Gene Therapy: A Lifesaving Advance for Infants with Rare Diseases Picture the heartbreak of learning your newborn has a rare, life-threatening genetic disorder with no proven cure. Now, imagine that within months, a customized therapy could rewrite that child’s dest... CRISPR gene therapy infant health liver disorders NIH research precision medicine rare diseases