Frequently Asked Questions
There are two paths. You or your physician can contact n-Lorem directly or one of the institutions in the Undiagnosed Diseases Network (UDN). In either case, the request will be reviewed by the Access to Treatment Committee of n-Lorem to determine the suitability of treatment with an experimental ASO. For more information on the process, click here.
To qualify for treatment with a personalized experimental ASO, each patient’s genetic mutation or change in gene function must be nano-rare meaning the same mutation is only found in approximately in 1 – 30 patients in the world. Each patient must live in the United States and have a diagnosis and the genetic cause must be defined. Patients must have a gene target in an organ that we target, and the patient must be cared for by a research physician and an institution (tertiary care center) experienced in managing Research INDs.
Unfortunately, at this time, n-Lorem can only treat patients who reside in the United States.
n-Lorem has been established to consider providing experimental antisense oligonucleotides (ASOs) treatments for genetically defined diseases caused by single gene mutations that are known to occur in 1-30 patients in the world.
Please have your physician reach out to us at [email protected] to discuss next steps.
Antisense technology, pioneered by Ionis, is very efficient technology for the discovery of new medicines. Antisense medicines are small bits of chemically modified genetic material to have the properties needed to be effective medicines that bind to target RNAs. Learn more
Modern genomic technology support rapid detail characterization of the genetic characteristics of individual patients, including identifying the gene involved in causing the disease and the type of change in the gene that the patient has.
As a nonprofit we rely on the generosity of our donors, supporters and partners. We have over 35 corporate partners who provide us with in kind and financial support across all areas of drug discovery, development and manufacturing. Without the generosity of all of our supporters, we would not be able to provide these medicines for free.
Sadly no. One of the functions of the Access to Treatment Committee is to determine if an experimental ASO medicine is appropriate for a patient.
n-Lorem is focused on creating individual treatments for patients in the United States with nano-rare diseases caused by genetic mutations that affect approximately 1-30 patients in the world. Diseases or conditions with broader patent populations are likely more suitable for treatment by other non-profit or for-profit entities, which we encourage you to explore with your physician.
A qualified investigator is an expert in the treatment of rare diseases who is experienced in performing clinical trials with new experimental medicines.
Once an application is received at n-Lorem, an initial check to make sure the minimum qualifications are met, our Access to Treatment Committee will review all the submission and make a recommendation to n-Lorem. It is critical that the patient’s physician be a qualified physician in the United States that is affiliated with a qualified institution.
Each patient program will have a unique timeline. For some programs, drug discovery is relatively straight forward and an optimal ASO can be identified relatively quickly. For others, the discovery process can be longer and in some cases, we may not be able to identify an optimal ASO to move forward in development. At a minimum, our programs will be approximately 18 months, but this timeline can slip significantly if there are any problems with ASO identification and development.
n-Lorem communicates regular updates to its submitting physicians. We encourage our submitting physicians to pass along these updates to their patients.
A qualified institution will typically be tertiary care centers that have experience in performing clinical trials with new experimental medicines.
n-Lorem collaborates with the research and/or treating physician and the institution where the patient will hopefully be treated. A collaborative engagement between all involved parties is a critical component of ensuring the most efficient path to treatment for the patient.
Both treatment evaluation and material transfer agreements are key components of the n-Lorem/institution relationship.
As a nonprofit that intends to provide experimental ASO medicines for free for life to nano-rare patients, n-Lorem has no intention of commercial development for its medicines. As such, n-Lorem’s limited resources are earmarked for the costs associated with discovering, developing and manufacturing experimental ASO medicines. Because of this, n-Lorem cannot pay institutions to cover the costs associated with providing data to allow assessment of the performance of the ASO medicine.
We are blinded to our patient’s identity, but if you would like to reach us, please email [email protected] and let us know who you are. We love to connect with our patients, hear and share (if approved) their stories and connecting them with our nano-rare community.
More questions?
Please reach out to us!
For additional information — please see our Frequently Asked Questions or reach out to us at [email protected].
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this alone
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one patient at a time
We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.
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