ISTANBUL, May 11, 2020 /PRNewswire/ — A fundraising campaign with a target amount of $2.4 million has been launched through GoFundMe crowdfunding site to save Nil’s life.
Nil Güleç was diagnosed with spinal muscular atrophy (SMA) type 2. To provide their daughter a healthier future, her parents are looking for ways to have access to a gene therapy called “Zolgensma.” The drug is approved for children under two years old and the clock is ticking against Nil as she has just turned 20 months on the 5th of May.
Zolgensma is designed to replace the missing or defective SMN1 gene with a functional copy that produces the SMN protein, thereby improving the function and survival of motor neurons. It seems that Zolgensma can save lives of those who are lucky enough to get it with a single dose of treatment. Yet the drug is currently the most expensive medicine in the world with a cost of 2.15 million US$. This price level is beyond the reach of her family.
“We, as her parents, without knowing that we are carriers of this disease, are broken. It is hard for us to breathe, talk or think. We need support during this fight and help of volunteers who can donate for our little daughter. We wish that Nil will be one of the lucky patients receiving this treatment and will be the hero of her own fairytale,” Nil’s parents remarked.
Nil’s parents have created a web site to reach the financing of the gene therapy. The name of the web site is originated from the donation campaign that has been initiated through “GoFundMe” crowd funding site in America: https://www.savingnil.com/en. Savingnil.com consists of artworks of esteemed painters, photographers, digital designers besides those who are engaged with art apart from their main profession. Artworks exhibited in this web site are all dedicated to Nil. The income gained from the sales on this site will be used for Nil’s treatment. This web site is another hope for Nil.
Spinal Muscular Atrophy (SMA) is a neuromuscular disease, which is manifested by a progressive loss of muscle strength that affects the ability to walk, swallow and breathe. And it is the number one genetic cause of infant mortality. It is caused by a mutation in the survival gene of motor neurons 1 (SMN1). This gene is responsible for the production of a protein that is critical for the nerves that control our muscles. Without this protein, these neurons cannot function properly and die at any given time.
For more information about donation, please go on https://www.gofundme.com/f/save-nil039s-life.
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SOURCE Save Nil’s Life