The goal is to raise enough funds ($2.1 million) to cover the costs of the gene-therapy treatment (Zolgensma) and additional expenses like travel, consultation fee and accomodation during her treatment abroad.
Robert just turned 1 this July, and needs help getting treatment for his disorder. The family lives in Norway, and the most effective treatment is currently not approved in his country, so she hopes to bring her boy here to the US for treatment. The problem is, the treatment he needs is one of the most expensive in the world! This account is to help Robert's family raise the funding needed for his treatment and associated travel expenses. Times are tough for everyone, but if you're able to spare some extra funds, please do so. Also, please share this story widely. Also, please be sure to read their story below. Anything you can give helps! We are trying to raise $2.1 million for Robert's rare medical condition, so any help you can give will go a long ways. BACKGROUND This is Robert (born on July 15, 2020. Robert has a rare genetic and degenerative disorder called SMA (Spinal Muscular Atrophy) type 2. SMA is a rare genetic disease that affects the central nervous system, voluntary skeletal muscle movement, and the peripheral nervous system (the connection between the central nervous system and the rest of the body). As a result, SMA affects basic motor functions such as walking, hand movements and feeding. After progression, the patients lose the ability to swallow, breathe and eventually die. SMA affects one in 10,000 live births and cannot be cured. However, today patients are given treatment options to prevent further progression, as any damage caused by the disease is irreversible. Robert developed like a normal child by age and achieved the appropriate milestones. After 11 months, Robert was diagnosed with SMA through genetic testing after we questioned his sudden regress of achieved developmental milestones and minimal use of his legs. Prior to this, we had never heard of SMA. Since the diagnosis, Robert has been put on Spinraza, a prescription drug administered as a spinal injection throughout a patient’s life. Spinraza increases the ability of the SMN2 gene (which is a back up of the SMN1 gene-the one that Robert is missing) to produce SMN protein which in turn improves motor functions and survival. Zolgensma is a new one time gene therapy treatment for kids under the age of 2. It REPLACES the missing SMN1 gene with a new, working copy that helps motor neuron cells work properly and survive but it’s currently the most expensive drug in the world at $2.1 million. It is NOT a cure since it does not reverse damage already caused by SMA before treatment. However, with supportive therapy, it has by far shown the best results. It is recommended that the earlier the patient receives the treatment, the better. We are fortunate and grateful to the Norway healthcare system for giving Robert Spinraza. However as parents, we cannot leave the approval of gene therapy in Norway to chance and hope that, it will be ready before Robert turns 2 years old. As any parent, we wish the best for our child, but even more so in regards to his health. Besides Zolgensma only having to be administered once, it has proven to be the most effective in clinical testing. OUR GOAL The goal is to raise enough funds ($2.1 million) to cover the costs of the gene-therapy treatment (Zolgensma) and additional expenses like travel, consultation fee and accomodation during her treatment abroad. By giving Robert this treatment, we hope to give his an almost normal childhood where he will be able to play with his big sister without any major obstacle. We are in a race against time to achieve our goal since this treatment is currently only approved for children under 2 years old. We would like to invite you to help Robert's family, friends and network create awareness around this fundraising by sending us your prayers, positive thoughts, contribute financially and/or share this invitation with more networks so that we may reach as many helpful souls as possible. Every single contribution means the world to us. We hope to get Robert treated as soon as we raise all the funds needed. $2.1 million will be used to buy the drug zolgensma. And any additional donations will be used for treatment at Boston Children's Hospital, travel and accommodation during our stay in the USA. Thank you so much for your kind donations!!
Can’t donate? Please share. Even a quick share on Facebook can help.
The average share raises $97.