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Impact Guru - Spinal Muscular Atrophy (SMA) CARE FUND

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$2,350.00 raised of $40,000.00 goal

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This project aims to provide financial aid to patients in India, affected by Spinal Muscular Atrophy Spinal Muscular Atrophy (“SMA”) is a neuro-muscular, progressive, genetic disease that robs people of physical strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat, or breathe. It is the number one genetic cause of death for infants. SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1). The SMA-affected children and adults are cognitively bright and are budding professionals in various streams, but are physically dependent on their family members, friends, or guardians for any of their daily basic mobility needs. There are 4 types of SMA, SMA Type 1 to Type 4, and many finer variations among these categories. Impact Guru identifies these patients who need financial support for their Hospital and related medical expenses, and supports them through crowdfunding and raising grants. These treatments are using USFDA-approved drugs currently available as follows: - Disease-modifying therapy: These drugs stimulate the production of SMN protein. Nusinersen (Spinraza®) is FDA-approved for both children and adults. Your provider injects the drug into the space around the spinal canal. - Gene replacement therapy: Children younger than two may benefit from a one-time intravenous (IV) infusion of a drug called onasemnogene abeparvovec-xioi (Zolgensma®). This therapy replaces a missing or faulty SMN1 gene with a functioning gene - A different medication, Risdaplam (Evrysdi®), helps adults and children older than two months. People take risdaplam daily by mouth (orally). We are currently raising funds to make medical drugs and treatment more affordable for those in need. Our goal is to support beneficiaries and provide them with access to life-saving resources that would otherwise be too expensive. With your help, we can make a significant impact on the lives of those who require medical assistance. Together, we can make a difference and ensure that everyone has access to the care they need. Thank you for your support in this important endeavor.
Impact Guru - Spinal Muscular Atrophy (SMA) CARE FUND

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