Overview

How the Foundation Works

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 n1-10 patients in the world. Diseases or conditions with broader patient populations are likely more suitable for treatment by other non-profit or for-profit entities, which we encourage you to explore with your physician.

With a seasoned leadership team and strategic partnerships, the n-Lorem Foundation provides the framework, funds and access for patients with ultra-rare diseases to receive experimental Antisense oligonucleotides (ASOs).

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Our Philosophy

The n-Lorem Foundation is dedicated to the principle that the individual is the indivisible unit of human value. To be optimally fair to the many, we must be fair to the individual. To be generous to the many, we must be generous to the individual. For the human collective to achieve its maximum potential, the individual must have the opportunity to create maximum value.

Patients with Ultra-rare Diseases Present Compelling Unmet Needs

Treating a patient who may be one of ten or the only person in the world with a specific genetic disease presents multiple challenges including:

  • Getting a proper diagnosis: Most often, genetic screening is needed to diagnose patients with ultra-rare diseases
  • Once diagnosed, understanding the genetic characterization: Once diagnosed, patients need to know if their disease is caused by a genetic mutation, and if so, what that mutation is
  • Identifying a physician specialist to provide proper treatment: Patients need a physician experienced with treating patients with ultra-rare diseases
  • Locating an experienced and properly equipped medical institution/facility: Patients should be treated at a medical institution or facility that is properly experienced and equipped to treat patients with their disease
  • Finding a company to develop an Individualized treatment: Patients need a medicine that is designed specifically to treat the genetic cause of their disease
  • Obtaining regulatory approval of the individualized treatment: Individualized treatment will usually require regulatory approval

The n-Lorem Foundation

Creating Individualized Treatments for Patients with Ultra-rare Diseases Using Antisense Technology

Traditional biotech and pharmaceutical companies are ill-equipped to address the needs of patients with ultra-rare diseases because the drug platform technologies they employ, such as small molecules, therapeutic proteins or antibody drugs, are not well-suited for creating individualized medicines. Costs and resources needed to develop new individualized medicines for ultra-rare disease patients with a broad diversity of diseases are prohibitive.

Advances in molecular biology now allow for relatively inexpensive DNA sequencing that supports the identification of the genetic causes of many diseases. Some ultra-rare diseases are caused by a single genetic defect unique to only one or very few individuals.

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