What diseases are candidates to be considered by n-Lorem?
n-Lorem has been established to consider providing experimental antisense oligonucleotides (ASOs) treatments for genetically defined diseases that are known to occur in 1-30 patients in the world.
What is antisense technology?
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.
How can you provide such medicines for free?
We collaborate with institutions such as those in the Undiagnosed Diseases Network (UDN) who determine the cause of the disease and Ionis to efficiently bring experimental ASOs to clinical investigators who can treat these ultra-rare disease patients.
We plan to provide these medicines for free because the efficiency of the antisense technology at Ionis and the generosity of donors.
How can I access treatment?
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.
Can all ultra-rare diseases be treated with ASOs?
Sadly no. One of the functions of the Access to Treatment Committee is to determine if an experimental ASO is appropriate.
What if there are more than 30 people in the world with the same genetic mutation?
n-Lorem is focused on creating individual treatments for patients in the United States with ultra-rare diseases caused by genetic mutations that affect approximately n1-30 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.
What is a qualified investigator?
A qualified investigator is an expert in the treatment of rare diseases who is experienced in performing clinical trials with new experimental medicines.
What is a qualified institution?
A qualified institution will typically be tertiary care centers that have experience in performing clinical trials with new experimental medicines.
What is a genomic analysis?
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.