Breakthroughs in rare disease and neurodegenerative disorder therapeutics are progressing rapidly. Stealth BioTherapeutics advances its Barth syndrome drug through regulatory efforts despite prior FDA rejection, while cortical organoid and exosome-based vaccines enter clinical evaluation. Novel eIF2B activators show promise for ALS treatments, complemented by gene therapies for systemic lupus erythematosus and inherited retinal diseases entering trials. Additionally, robotic-assisted pediatric spine surgery and laser-free corneal reshaping offer innovative clinical interventions. These developments signify a broadening toolkit to address complex disorders with unmet medical needs.