Rare Genetic Disease Program
Patients, of all ages, with rare genetic diseases, familial Alzheimer's, familial Parkinson's, and ALS. (For familial Alzheimer's and Parkinson's, please get genetic testing done before signing up).
This program consists of a stem cell biobank made up of blood samples from patients. We isolate the stem cells from the blood that contain the genetic defect and use them for drug testing. If a drug cures the disease in the cells, it has the chance of entering a clinical trial and helping patients.
We will notify you if a drug cures the disease in your cells. That said, we CANNOT guarantee that we will find a cure for your disease.
Eligible? Interested in Joining? Following These Steps:
2. Fill out the form and submit.
3. Fill out and send the Consent to Release Form. Download it here.
Participation is voluntary. There is no charge to participate. Your medical records will remain confidential.
When Donating a Blood Sample:
When we are ready to conduct research on your disease, we will notify you and send a kit to your physician. Please set up an appointment with your doctor and make him/her aware of this program.
You will have to sign the consent form. You can download and print it here. Please have the form filled out before attending your appointment. At your appointment, your physician will draw the blood sample. Do NOT draw your own blood yourself. Let the professional do it.
Other Purposes of the Patient Registry:
Research advances and clinical trials are not possible without adequate patient participation, and therefore it is critical to recruit as many patients as possible. We will use the Patient Registry to help us recruit patients for clinical trials. If a researcher or organization asks us for assistance with patient recruitment for a clinical trial that involves your disease, we will also help them by notifying you about the trial.