The parents of three-month-old Leon from Tashkent have appealed to the residents of Uzbekistan for help for their son, who has been diagnosed with Type 1 Spinal Muscular Atrophy (SMA). This rare genetic disease causes gradual muscle atrophy.
The only way to stop the progression of the disease may be the use of the drug Zolgensma, which costs about two million dollars. It is critically important to administer this drug before the child reaches six months of age.
The boy's mother, Anastasia, reported that the diagnosis was made late, leading to irreversible damage to his body. Currently, Leon is receiving the drug 'Risdiplam,' which is provided free of charge by the state, but it only slows down the disease and requires lifelong treatment.
Anastasia described the family's difficult situation: 'Every day of ours is a struggle. Instead of walks, first smiles, and peaceful nights, our life has turned into endless home resuscitation. Mechanical ventilation (approx.), mucus removal, tube feeding, constant saturation monitoring, and the fear that at any moment my son's condition could worsen. We dream of only one thing—that Leon can stop living among medical equipment and be just a child.'
According to specialists, Leon has good prospects for receiving gene therapy. For this, the boy needs to undergo a special blood test in Moscow that will confirm the possibility of administering the drug. Doctors also emphasized that the lower the child's weight at the time of starting Zolgensma treatment, the better the body will tolerate the therapy and the lower the risk of serious side effects, so postponement is unacceptable.
The family cannot raise the required sum of two million dollars on their own. The mother expressed hope in the responsiveness of the people, stating: 'No child should die from a disease that modern medicine can already treat.'
To ensure transparency in fundraising, the parents decided that if Leon does not receive Zolgensma for any reason, all collected money will be transferred to another child with SMA. They also promised to regularly publish reports on income and expenses on Leon's Instagram page and on the parents' personal accounts.
Anastasia additionally mentioned that she has compiled all of her son's medical documentation in a Google Doc, the link to which is also available on Leon's Instagram page for review. In conclusion, she called on everyone who can to help, adding that even reposting the story can attract someone who can give Leon a life independent of constant therapy.
The article also provides information on the types of SMA: Type I, diagnosed in infants under six months, is the most critical because children cannot sit, and the risk of respiratory paralysis rapidly increases. Type II appears later, between 6 and 18 months, allowing children to sit, but they do not walk. Milder forms—Type III and Type IV—only cause gradual muscle weakness and have little impact on life expectancy.



