Qualifications for Treatment
Before submitting an application, you should review our qualifications to see if you meet the criteria for treatment.
For Patients
Do I qualify?
To qualify for treatment with an experimental ASO, you must have a diagnosis, the genetic cause must be defined, and the patient must be cared for by an investigator and related institution experienced in managing Research INDs. Therefore, we recommend:
Qualifications
- Patients must either live in the United States or have an identified research physician in the United States and have the ability to stay in the United States during the treatment period and evaluations per the treatment protocol.
- A 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 have a diagnosis and should have a full genomic analysis
- The type of change in genetic function must be understood, often this means that the patient is referred to a geneticist.
- Each patient must work closely with their research physician, as it is critical that each patient is under the care of an experienced research physician with the knowledge and experience to submit and manage a Research IND.
- Each patient’s research physician must be at an institution (tertiary care center) capable of expeditiously supporting a Research IND.
- Please consider working with investigators in the Undiagnosed Disease Network if any of the above elements are unavailable to you.
- Please understand that ASO technology, like all drug discovery technologies, has limits and we may not be able to help every patient.
For Physicians
Does my patient
qualify for treatment?
To qualify for treatment with a personalized experimental ASO, each patient must have a diagnosis, the genetic cause must be defined, and the patient must be cared for by a research physician and an institution (tertiary care center) experienced in managing Research INDs. Therefore, we recommend:
Qualifications
- Patients must either live in the United States or have an identified research physician in the United States and have the ability to travel to the United States for treatment and evaluations per the treatment protocol.
- A 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.
- A patient’s genetic mutation or change in gene function must be nano-rare, meaning it affects approximately n1-30 patients in the world
- Each patient must work closely with their research physician and institution.
More questions?
Please reach out to us!
For additional information — please see our Frequently Asked Questions or reach out to us at [email protected].
We cannot do
this alone
Together we are changing the world—
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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