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At N1, our purpose is to restore lives and make a positive impact on the people we serve. Therefore, we prioritize the well-being of those we share this world with and have a social contract with the world that reflects our commitment to this cause. You can view our social contract on our website at https://nell-one.com/who-we-are/social-contract/.
While our current focus is on creating a pharmaceutical solution for acute respiratory distress syndrome (ARDS), we seek development partners for various indications. In addition, we have a particular interest in rare diseases, as we understand the unique challenges and, at times, feelings of isolation and hopelessness faced by those who suffer from conditions that don’t always receive the attention they deserve. We keep these brothers and sisters in our prayers and promise to keep advancing NV1, hoping it may someday help. At N1, we always look to do the next right thing.
Rare Disease Day is a significant opportunity to raise awareness about individuals with rare diseases struggles. Rare diseases affect a small but significant portion of the population, and many individuals and families need support and resources to manage their conditions.
Watch the video here: http://bit.ly/3ZcYflG
In line with this mission, we are exploring the potential of our proprietary version of the NELL-1 protein, NV1, as a potential treatment for rare neurological diseases. It’s important to note that our research on NV1 is in the thesis stage and unavailable as a drug and that none of the following statements have been reviewed by the FDA. Nevertheless, we share this information as an invitation to potential collaborators and for educational purposes.
At N1, we are dedicated to positively impacting the lives of those affected by rare diseases and always seeking innovative solutions to make a meaningful difference. We seek partners to join us in doing the next right thing.
NELL-1 (Neural Epidermal Growth Factor-Like 1) is a protein that has shown promise as a potential treatment for rare diseases. NELL-1 is involved in developing and maintaining the nervous system, and research has suggested that it may have therapeutic potential for various neurological conditions.
One of the most promising applications of NELL-1 is in treating rare genetic disorders that affect the nervous system, such as leukodystrophies and lysosomal storage disorders. Leukodystrophies are a group of rare genetic disorders that affect the myelin sheath, a fatty substance that covers nerve fibers and facilitates nerve transmission. Lysosomal storage disorders are a group of rare genetic disorders that result from the accumulation of toxic substances in lysosomes, the cellular organelles responsible for breaking down and recycling waste products.
Research has shown that NELL-1 may promote the growth and differentiation of oligodendrocytes, which produce myelin in the central nervous system. In addition, NELL-1 can potentially reduce neuroinflammation and oxidative stress, common neurodegenerative disorders. These properties of NELL-1 make it a promising candidate for treating leukodystrophies and lysosomal storage disorders.
Another potential application of NELL-1 is in treating rare neurodegenerative disorders, such as Huntington’s disease, Amyotrophic lateral sclerosis (ALS), and Alzheimer’s disease. These disorders are characterized by the progressive loss of neurons and the accumulation of toxic protein aggregates in the brain. In addition, NELL-1 has been shown to potentially reduce microglial activation and promote the clearance of amyloid-beta, a key pathological feature of Alzheimer’s disease. These findings suggest that NELL-1 may also have therapeutic potential for other neurodegenerative disorders.
Huntington’s disease affects 1 in 10,000 people in the US, characterized by cognitive, behavioral, and motor function deterioration. ALS affects 5,000 people in the US each year and causes muscle weakness, disability, and eventually death. Both conditions significantly impact individuals and their families, with no current cure but management options available.
Alzheimer’s is not considered a rare disease, as it affects a significant portion of the population, with an estimated 6.2 million people in the US affected by it. However, research on Alzheimer’s disease can have important implications for other neurodegenerative diseases, such as ALS and Huntington’s disease. While these diseases differ in their specific symptoms and causes, they share many common underlying disease mechanisms, such as the accumulation of abnormal proteins in the brain. By studying Alzheimer’s disease, researchers may be able to uncover new insights and potential treatments for other neurodegenerative conditions.
NELL-1 | NV1 are promising proteins with therapeutic potential for various common and rare diseases. Its potential ability to promote the growth and differentiation of oligodendrocytes, reduce neuroinflammation and oxidative stress, and modulate microglial activation makes it a promising candidate for the treatment of rare genetic disorders that affect the nervous system, as well as neurodegenerative disorders. While more research is needed to fully understand the mechanisms by which NELL-1 | NV1 exerts its therapeutic effects, its potential as a treatment for rare diseases is an exciting area of investigation.
For Partnership Opportunities, contact Bill@Nell-One.com.
On National Rare Disease Day, we encourage you to donate to non-profit organizations that support rare disease research and provide resources and support for those affected by these conditions. These organizations have no affiliation with NellOne but are doing important work in the field. Donating to these organizations is an excellent way to support individuals and families affected by rare diseases and positively impact the fight against these conditions.
- The Huntington’s Disease Society of America – A non-profit organization that provides support, education, and advocacy for individuals with Huntington’s disease and their families. They also fund research into Huntington’s disease and related conditions. https://bit.ly/SupportHDSA
- The ALS Association – A non-profit organization that provides support, education, and advocacy for individuals with ALS and their families. They also fund research into ALS and related conditions. https://donate.als.org/give/287064/#!/donation/checkout?c_src=jumppage
- The Hereditary Disease Foundation – A non-profit organization that supports research into Huntington’s disease and related conditions. They also provide support and resources for individuals and families affected by these diseases. https://www.hdfoundation.org/donate-today
- The Muscular Dystrophy Association – A non-profit organization that supports research and provides resources and support for individuals with ALS and other neuromuscular disorders. https://mda.donordrive.com/index.cfm?fuseaction=donate.event&eventID=824