A wave of support has emerged online for a Thai child diagnosed with Aromatic L-amino acid decarboxylase deficiency (AADC Deficiency), a rare genetic disorder affecting only about 130 people worldwide.
The one-year-old child, identified only as Akin, is receiving treatment at King Chulalongkorn Memorial Hospital in Bangkok, where the family faces staggering medical expenses of up to €3 million, (105 million baht) for specialised gene therapy.
The condition hampers the ability of the body to produce vital neurotransmitters such as epinephrine, dopamine and serotonin, significantly limiting the child’s daily life.
Patients with AADC deficiency have little to no functional motor movement and never meet developmental milestones. They have the need for lifelong care and are at a high risk of early death in the first decade of life.
The necessary treatment involves the gene therapy regime Eladocagene exuparvovec, which comes with a high price tag. This therapy is accessible only outside of Thailand.
The first three children diagnosed with the condition have been able to participate in drug trials, but Akin has not been so fortunate, leading the family to seek other treatment options.
Due to the necessity of self-financing, King Chulalongkorn Hospital has negotiated with a pharmaceutical company to reduce the cost from €3.6 million to €3 million. Akin's father, who is a pharmacist, is doing everything he can to secure the needed funds.
The father started fundraising after learning that the trial queue in the United States was full with over 20 cases, as it only accepts 15 participants. China, which is also conducting trials, limits its trials to domestic patients.
On Jan 4, the father posted on Akin's fundraising Facebook page that his child is currently 15 months old. He said treatment should begin between 18 and 30 months of age to achieve the best results.
In a show of solidarity, internet users have been sharing a GoFundMe campaign initiated by the Thai Red Cross Society and the Teach Rare Foundation — a group founded by Richard E Poulin III and Judy Wei following their daughter's own AADC deficiency diagnosis.
Contributors are encouraged to visit www.gofundme.com to support Akin and his family in their time of need.
