“I am highly optimistic that this gene therapy can represent an efficacious and safe therapy for patients with schwannoma – whether associated with NF2, schwannomatosis or sporadic tumours”.
Gary J Brenner, MD, PhD, Associate Professor, Harvard Medical School. Director, Massachusetts General Hospital
What is Gene Replacement Therapy?
video provided courtesy of Avexis
We are just starting, we need you to start the toxicology study!
Mission: We are a non-profit organization (Tax-ID 83-0940046) seeking a cure or preventative treatment for NF2 by vigorously supporting and advancing existing and next-generation biomedical technologies, such as gene therapy and immunotherapy.
Vision: We envision a world where those living with NF2 will have access to cutting-edge solutions to live full and productive lives, free from daily physical and emotional pain or suffering.
Why is rare disease research important?
- Over 7,000 rare diseases have been identified.
- While each disease on its own is rare, 1 in 10 people are living with a rare disease. Every treatment that is developed helps to pave the way for other treatments.
- Many cancers and benign tumors show mutations in the NF2 gene. A therapy for NF2 would also help in treatment of those cancers. In addition to curing NF2, help us cure sporadic vestibular schwannomas (formerly know as acoustic neuromas), meningiomas, breast cancer, prostate cancer, colorectal cancer, and mesothelioma, amongst many others.