Seeking World Premiere ‘RARE’ unveils what stands between the families fighting rare disease, hoping for treatments, and the emerging groundbreaking therapies available. RARE highlights the neglect faced by families battling rare diseases within the broader healthcare system. Due to the rare nature and thus limited profit margin of these conditions, large pharmaceutical and biotech companies often turn a blind eye towards developing treatments. Unlike more common diseases like cancer, parents of children with rare diseases are burdened with the responsibility of raising funds and spearheading drug development to save their children. Far from succumbing to their dire situation, these families are channeling their energies into becoming influential advocates and pioneers, pushing forward scientific advancements such as gene therapy and antisense oligonucleotides (ASO). Their fight is not just for their own children but for future generations diagnosed with these diseases. The 6 families we profile are superheroes, making the impossible possible for their child, and an inspiration for anyone facing life’s challenges.