Our mission is to raise awareness and fund research towards the development of life-saving treatments and a cure for NUBPL, a mitochondrial complex 1 deficiency disorder.
NUBPL is a progressive neurodegenerative disease that affects children. There are zero FDA approved treatments for this disease. Patients with NUBPL present clinical symptoms between the ages of 3-24 months, including onset neurological symptoms: cerebellar dysfunction – progressive cerebellar & pons hypoplasia, global developmental delay, inability to walk, ataxia, nystagmus, and speech articulation difficulty.
Researchers are studying NUBPL to discover a treatment, and hopefully, a cure. Every dollar donated is filled with hope. Help keep hope alive for their future by making a tax-deductible donation today.