Application for Treatment

n-Lorem is focused on creating individual treatments for nano-rare patients who have a gene mutation that affects approximately 1 to 30 patients with the same single gene mutation worldwide. 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 before submission.

Please read this before
you submit:

  • n-Lorem only allows research physicians to submit an application to treat for their patients.

    There is a substantial amount of information concerning the patient that needs to be submitted with the application in order for n-Lorem and the Access to Treatment Committee to make decisions concerning the potential to treat each patient with an experimental ASO.

    Included in the submission are:

    • Diagnosis
    • Characterization of the mutation
    • Functionalization of the gene
    • Clinical pathophenotype
    • Organs affected
    • Primary manifestations of the mutation to be treated
    • Other disease manifestations
    • Goals of the treatment (e.g. what symptoms do you expect to improve)

    Read more about our criteria and
    qualifications:

    Qualifications for Treatment

  • ASO technology is versatile and broadly useful but has limits. Please be aware of the limits of the technology.

    Strengths and limitations of ASO technology:

    ASO technology is validated, versatile (multiple post-RNA binding mechanisms), effective in many organs at low doses, deliverable by many routes of administration, and active both systemically and locally. Moreover, ASO medicines of each specific chemical class behave similarly, facilitating dose and schedule selection. However, as is the case for all drug discovery technologies, ASO technology has limits. ASO technology cannot replace genes, therefore, true null mutations may be better treated with other therapeutic approaches. Compound heterozygous mutations are unlikely to be treatable by ASOs, or if the gene in question serves a vital function, then reducing the transcript from that gene may be too risky.

Patient Submission Form Portal

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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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