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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 heart 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 dilated cardiomyopathy. NELL-1 is involved in the development and maintenance of the heart and has been shown to promote the growth and differentiation of cardiac progenitor cells, which are involved in the repair and regeneration of heart tissue.
Research has shown that NELL-1 can improve cardiac function in animal models of dilated cardiomyopathy. In one study, mice with dilated cardiomyopathy were treated with NELL-1 and showed significant improvements in heart function and structure compared to control mice. The researchers also found that NELL-1 treatment increased the number of cardiac progenitor cells and reduced fibrosis, a hallmark of heart disease.
NELL-1 has also been shown to have potential as a treatment for other rare heart diseases, such as arrhythmogenic right ventricular dysplasia/cardiomyopathy (ARVD/C) and hypertrophic cardiomyopathy (HCM). ARVD/C is a rare genetic disorder affecting the heart’s structure, while HCM is a genetic condition that causes the heart muscle to thicken. Both conditions can lead to heart failure and sudden cardiac death.
ARVD/C and HCM are two rare genetic heart conditions that affect an estimated 1 in 5,000 individuals in the United States. ARVD/C affects the right ventricle of the heart and can lead to abnormal heart rhythms, while HCM is characterized by an abnormally thickened heart muscle that can cause difficulties in pumping blood. Both conditions can increase the risk of sudden cardiac death and may have serious consequences for those affected. Therefore, individuals with a family history of either condition must undergo genetic testing and regular heart screenings to help reduce the risk of complications and improve outcomes.
In a study of mice with ARVD/C, NELL-1 treatment improved heart function and reduced fibrosis and inflammation in the heart. Similarly, in a study of mice with HCM, NELL-1 treatment reduced the thickness of the heart muscle and improved cardiac function.
NELL-1 / NV1 are promising proteins with therapeutic potential for rare heart diseases, such as dilated cardiomyopathy, ARVD/C, and HCM. Its potential to promote the growth and differentiation of cardiac progenitor cells, reduce fibrosis and inflammation, and improve heart function makes it a promising candidate for treating these conditions. 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 heart 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.
1. National Organization for Rare Disorders (NORD) – NORD is a non-profit organization dedicated to improving the lives of people with rare diseases. They provide support, education, and advocacy for patients and families affected by rare diseases, including those that affect the neuro, cardio, and pulmonary systems. Donations can be made on their website: https://rarediseases.org/donate-nord/
2. Global Genes – Global Genes is a non-profit organization that works to eliminate the challenges of rare diseases by providing resources and support to patients and families affected by rare diseases. They focus on advocacy, education, and awareness building for rare diseases, including those that affect the neuro, cardio, and pulmonary systems. Donations can be made on their website: https://globalgenes.org/other-ways-to-support/
3. Rare Disease United Foundation – The Rare Disease United Foundation is a non-profit organization that provides advocacy, support, and education for patients and families affected by rare diseases. They focus on building awareness and research for rare diseases, including those that affect the neuro, cardio, and pulmonary systems. Donations can be made on their website: https://rarediseasefoundation.org/pages/donate
4. The Hypertrophic Cardiomyopathy Association – A non-profit organization that provides support, education, and advocacy for individuals with HCM and their families. They also fund research into HCM and related conditions. https://4hcm.org/donate/
5. The ARVD/C Foundation – A non-profit organization that provides support, education, and advocacy for individuals with ARVD/C and their families. They also fund research into ARVD/C and related conditions. https://sads.org/donate/