The author of the article is the mother of a child with a rare disease.[1] Personalized therapies have long been promised but unavailable to families with rare diseases.[1] The first drug for spinal muscular atrophy (SMA) became available in 2017.[1] In January 2021, the European Medicines Agency (EMA) approved a new SMA drug for patients who are not suitable for current treatment.[1] Teams of experts have been built in Slovakia, diagnostics have improved, and a pilot project for SMA screening is being prepared.[1] Half of the patients with rare diseases are children, but patients live to an older age thanks to the knowledge of the diseases and non-pharmacological support such as physiotherapy or music therapy.[1] Personalized medicine involves treatment tailored to the patient's genetic profile.[1][7]