Hammad Ansari is a 12 year old boy who's unable to walk. He has Duchenne Muscular Dystrophy. The treatment, recently FDA approved, is extremely costly and Hammad's family is unable to afford it. Doctors say he's got a life expectancy of upto 17 years max if not cured.
DMD is the most common type of muscular dystrophy, a rare genetic disorder characterised by progressive muscle deterioration and weakness. It is caused by mutations in the DMD gene which results in an absence of dystrophin, a protein found in muscle fibres. The first symptoms are usually seen between three and five years of age and worsen over time. The disease occurs in approximately one of every 3,600 male infants worldwide and can, in very rare cases, affect females.
The US Food and Drug Administration has conditionally approved Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. This is the first FDA-approved targeted treatment for the eight percent of DMD patients with this type of mutation.
This treatment is extremely expensive and Hammad's family are unable to afford it. Without the treatment, Hammad can only live upto 17 years — 19 if he's lucky. Please help Hammad walk again. Help him live longer.