Help Lucas to receive Risdiplam Treatment

Lucas was diagnosed with Spinal Muscular Atrophy (SMA) type 1. SMA is the leading genetic cause of infant death. If left untreated, SMA Type 1 leads to death or the need for permanent ventilation by the age of two in more than 90% of cases. SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing, and basic movement. It is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression. This is especially critical in SMA Type 1, where motor neuron degeneration starts before birth and escalates quickly. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities. It is a rare neuromuscular disorder that results in the loss of motor neurons and progressive muscle loss. To have a better future, Lucas must receive genetic therapy called Zolgensma. The Zolgensma cost $2.1million, Zolgensma approval only covers children under 2 years old. There's another available treatment, nusinersen (Spinraza) – a medicine that targets the backup copy of the faulty gene, given as an injection into the spine, every few months. Risdiplam (Evrysdi) – a medicine that targets the backup copy of the faulty gene, given as a liquid once a day. (400,000 per bottle)

Lucas is 6 months old. It’s a fight against time.

Your help and donation are a big help for Lucas Kaven Gungon Malig.