Spinal muscle atrophy treatment for Ali Balaghi

Hi my name is Hussein. I’m raising funds for

Ali BALAGHI, born on January 17th, 2022, is a 9 months old toddler with spinal muscular atrophy (SMA) confirmed genetically by the presence of homozygous deletion of the SMN1 gene with detection of three copies of SMN2 gene. The disease started at 4 months with progressive generalized hypotonia predominantly proximal contrasting with preserved alertness. Ali has no history of hospital admission for respiratory distress and is undergoing physical

therapy. He is an excellent candidate for gene therapy (brand name : Zolgensma®) which is the only curative available treatment for SMA. It is needed urgently before Ali’s condition worsens. Unfortunately, this treatment option is not available in Lebanon therefore the parents need to travel abroad seeking for help.

In the meantime and in addition to the supportive treatment, the available approved treatment for oral use (Evrysdi ®) must be given as early as possible for Ali to have a better motor function and to minimize the orthopaedic complications and the respiratory problems that affect his

quality of life as well as his life expectancy.