In honor of Neurofibromatosis Awareness Month, and Neurofibromatosis Type 2 Awareness Day on May 22, we are proud to spotlight one of our parent-founded rare disease partners to help raise visibility about rare diseases and their significant impact on individuals and families.
Neurofibromatosis Type 2 (NF2) is a rare genetic disorder that results in the development of tumors throughout the nervous system. Common symptoms include hearing loss, tinnitus, balance problems, vision impairment, and can also include headaches, facial weakness, numbness or weakness in limbs, and seizures. There is currently no cure for NF2, but treatments can help manage symptoms and improve quality of life by slowing the progression of the disease. Treatment options may include surgery, radiation therapy, chemotherapy, and supportive therapies like hearing aids and rehabilitation. Ongoing research and clinical trials are exploring new treatments, including gene therapy and immunotherapy, to find a cure or preventative treatment for NF2.
Dr. Nicole Henwood founded the non-profit NF2 BioSolutions, and Merlin Therapeutics, a for-profit therapeutic company, when her son was diagnosed with NF2. Genezen is partnering with the organizations to help advance a novel gene therapy to slow the progression of the disease.
On Rare Disease Day, Dr. Henwood agreed to a video interview with our Associate Director of Marketing & Communications, Meg Bor – who is co-leading our Rare Disease Partner Network program. Watch the full interview below to learn about Dr. Henwood’s experience with her son’s diagnoses and why she chose to work with Genezen on the production of her organization’s novel therapy.
“In our society the people who are suffering with the disorder are often the ones left to figure out the cure. We’re left to cure ourselves. And that’s for every disorder, not just NF2. But why is that? Our mission is to change that. We won’t give up.”
On choosing to work with Genezen in the development of their NF2 gene therapy, Dr. Henwood says it came down to being confident in the science and the relationships that she and her team fostered with Genezen staff and leadership. She notes that both things are equally important. “Of course, the science and the technical experience and the knowledge of the team – and knowing a few members of the team before Genezen was even in existence – gave us confidence. The level of support, the passion and knowing that the team sees our mission the same way we do. That we have to get out there and put an end to these diseases. Somebody’s gotta do it, so why not us.”
Dr. Henwood further comments, “Every step, every time any disorder, be it rare or not, gets to a treatment, you’re helping the next ones that are diagnosed…Every single one that we get closer to the finish line is going to make the next rare disease…easier and more streamlined, and a better process for the future. We need passionate people that are going to stick with it and walk the path to the end of the homerun.”
Support NF2 BioSolutions in honor of NF2 Awareness Day
NF2 Awareness Day is an opportunity to spread awareness and fundraise. NF2 is still unfortunately misunderstood. For example, just because it is the second NF disease, it is not a subclassification, not less severe. In fact, NF and NF2 are distinct disorders. They were named by similar clinical findings before DNA was a known thing. Spreading awareness about NF2, what it is, how it affects people, that there is a path forward – this is the purpose of NF2 Awareness Day.
Says Dr. Henwood, “The day is an opportunity to teach at least one other person what NF2 is. Any donations are super appreciated and welcome. A little goes a long way in this world.”
As problem solvers who appreciate other problem solvers, we are proud to partner with Merlin Therapeutics and support NF2 BioSolutions.
Steve Favaloro, Chairman and CEO at Genezen commented, “Genezen’s partnership with NF2 BioSolutions underscores our commitment to the rare disease community and demonstrates our CURES values. It is our sincere mission to partner with foundations like NF2 BioSolutions and grow with them as they advance from early-stage trial to commercial treatment.”
To learn more about NF2 BioSolutions and support their efforts to find novel therapies to cure NF2, please visit https://nf2biosolutions.org/
