Help Cure NBIA-MPAN

A personal campaign sponsored by Tuba Alam

April 27, 2019

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Ryan was recently diagnosed with NBIA (neurodegeneration with brain iron accumulation). NBIA is a group of inherited neurologic disorders in which iron accumulates in the basal ganglia (the portion of the brain that controls movement and other critical functions). It’s a progressive neurodegenerative disease that leads to patients developing the inability to walk, dementia, Parkinson’s disease, loss of ability to swallow, problems with vision and etc. There are different types of NBIA, including PKAN, MPAN, BPAN, and more. Since 2001, there have been around 500 known cases of NBIA worldwide. Fewer than 100 of those have been MPAN, the type of NBIA that Ryan has. MPAN is caused by a single defective gene that is related to mitochondrial function. There’s currently no cure for any form of NBIA, including MPAN, and all doctors can do is treat the patients’ symptoms. Since this condition is so rare there is virtually no money for research.


Ryan is 15 years old now and we noticed his gait changed as he got taller (around late elementary school). After years of appointments with different doctors, physical therapy, aqua therapy, occupational therapy, and private gym trainers, the iron showed up on his most recent MRI of the brain. The neurologists immediately ordered genetic testing to confirm what they were suspecting – the MPAN form of NBIA. If you ever meet Ryan, you will know how special he is. We can’t say enough about how strong, resilient, and incredible this child is given everything he has been through in the past 15 years. He is just amazing! There isn’t a day that goes by that Ryan doesn’t have a smile on his face, even with his condition. He is aware of the condition he has and the effects it is having on him, but he doesn’t let it bring him down. Instead he tells us that he will fight it and reminds us to be strong and have faith. He has been through many hospital visits, the worst of which was due to an accident last year. His weak legs caused him to fall through a glass shower stall. He got over 300 stitches and staples all over his face, head, and body. Even at the hospital he amazed the doctors with the smile on his face. When the doctors and hospital staff asked what they could do for him, he responded with, “My mom is really worried.  Can you please take care of her?” Even in his hardest times he is thinking of others.


Ryan is very excited about raising money to help find treatments or a cure for MPAN. He wants to get better and help others get better as well. We are working with a wonderful group of doctors and researchers at Children’s National Hospital in Washington, D.C. to assist with research efforts. All money raised will go to support both clinical and laboratory research-based investigation into the clinical presentations and mechanisms underlying neurometabolic diseases, including MPAN-NBIA, and development of new therapies to treat these disorders. We ask that you please help us raise the money needed to research MPAN-NBIA so kids like Ryan have a chance to live a normal life. All they want is to be treated like any other kid and to be able to do the things other kids can do. You can help us make that possible.



Thank you to our supporters!
 

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