Netizens unite to help Thai child with rare disease
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By: Rujapa (Kayla) Ketvoravit
A one-year-old boy, Akin, was diagnosed with an extremely rare condition Aromatic L-amino acid decarboxylase (AADC) deficiency. This condition is a rare genetic disorder that affects only about 130 people worldwide. Akin is receiving treatment at King Chulalongkorn Memorial Hospital in Bangkok. His family is facing expensive medical expenses up to 105 million baht for specialized gene therapy. The condition hinders the body’s production of vital neurotransmitters such as epinephrine, dopamine and serotonin, which limits the child’s everyday life.
Patients who have AADC deficiency have very little to no functional motor movement and they are unable to meet developmental milestones. They need to have lifelong care and often have a higher risk of early death in the first decade of their life. The therapy that is a necessary treatment is only accessible outside of Thailand. It involves the gene therapy regime Eladocagene exuparvovec, which comes at a high price.
The first three children who have been diagnosed with this disorder have been able to participate in drug trials. Akin’s family does not have enough money, making the family seek for other treatment options. Due to the necessity of self-financing, King Chulalongkorn Hospital has negotiated with a pharmaceutical company to reduce the cost of the treatment from €3.6 million to €3 million.
Akin‘s father decided to start fundraising after learning that the trial queue in the U.S was full with over 20 cases. It only accepts 15 participants from China, which is also managing trials.
On January 4, his father posted on Akin’s fundraising Facebook page that Akin is currently 15 months old. He also said that the treatment should be between 18-30 months of age to get the best results. In a show of solidarity, many people decided to share Akin’s story to help raise money for his treatment.