SMA I (Werdnig-Hoffmann's disease) is an unusual and genetic neuromuscular disease that affects hundreds of children in the world every year. SMA1 is the most common form. The disease is caused by an SMN1 gene which results in the loss of motor neuron required in turn affecting muscle functions such as breathing, swallowing ability and mobility. The classic form, SMA I, is the most difficult variant that occurs in infants.
On May 24, 2019, the US FDA approved a new gene therapy from Novartis for the treatment of spinal muscle atrophy. The special thing about the drug Zolgensma is that it costs 2.1 million dollars, which makes it the world's most expensive medicine.
Zolgensma repairs the genes so that the protein that is missing in patients with the hereditary disease can begin to produce and is a one-time treatment.
Recently, a new drug SPINRAZA has emerged, a so-called antisense oligonucleotide, which, by binding to a specific region in SMN2, increases the level of full-fledged SMN protein. Studies in children with SMA have recently (2017) shown that SPINRAZA has positively affected the course of the disease. The drug is given by repeated injections into the spinal fluid (lumbar puncture).
Muhammed Ali was born in Antalya, Turkey on 2019. 12. 23 and was diagnosed as SMA1 on 2020. 07. 28. He cannot walk or sit and eat and drink, so is completely dependent on all help. A few months ago, he was admitted to the intensive care unit where he underwent a tracheostomy due to. swallowing difficulties But now he is being cared for at home and needs your help to get treatment to keep his joy alive.
Your valuable support means a lot
Research shows that this treatment can be crucial for the child's health and development and therefore early treatment is extremely important for M. Ali's health condition. Now there is hope that M. Ali can live and does not have to leave the world too soon. Show your support and compassion for hope in the world continues that we can only together succeed in making him feel good. Thank you for your support and help!
- Sevgi Dogan
- Viona Isufi
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