How Ireland's Case and Your Support Have a Bigger Impact
Rare Disease Problem
- Number of people with rare diseases: Over 300 million people worldwide have rare diseases. In the United States, 1 in 10 people have a rare disease, which is about 25–30 million Americans.
- Number of rare diseases: There are over 6,800 rare diseases, and 72% of them are genetic.
- Treatment availability: Only about 5% of rare diseases have FDA-approved treatments.
FDA Approved Clinical Trial
N-Lorem and a Principal Investigator will submit a Research Investigational New Drug (IND) application to the FDA on Ireland's behalf. Upon regulatory permission and IRB approval, an investigator-led clinical trial is initiated and Ireland will receive her personalized medicine. n-Lorem and a research physician will collect data to measure treatment goals. This process will continue throughout the length of treatment.
Source: www.nlorem.org
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Impact on Rare Disease and CACNA1A
As an open-access foundation, n-Lorem will work closely with the physician to publish preclinical and clinical data in peer-reviewed and scientific forums. This data could impact how the medical community thinks about disease and the link between genotype and phenotypes and also how disease phenotypes shift throughout the lifetime of a patient.
If n-Lorem has a therapy available for a nano-rare patient that has the potential to help other patients with similar mutations, those families or a physician of a potential patient may contact n-Lorem for information on that program.
Source: www.nlorem.org