RARE Global Advocacy Alliance Member Spotlight: Geoffrey Gee

February 24, 2023

Geoffrey Gee, Member Spotlight, Feb 2023

Geoffrey Gee

Organization Name
Neuromuscular Disease Foundation

Disease Affiliation
GNE Myopathy

Organization Mission
We work to enhance the quality of life for those living with GNE Myopathy by funding critical research focused on treatments and a cure through The International Gene Therapy Development Program (IGTDP) and by advocacy, education, and outreach.

What led you to the rare disease community?
I have had many years working in the non-profit world. When I was approached in late 2021 to become NDF’s Executive Director, I knew I could offer a great deal to the organization based on my legal, financial, and fundraising background and experience. I have MS, and this often starts with a foot drop, much like GNEM. I now need to rely on a cane, and I feel that I bring a different perspective to the ED position as I more closely understand our patients’ challenges.

What do you think are the areas that are lacking in the community (specific to your org or in general)?
With approximately six patients in a million suffering from GNEM, it’s challenging to create awareness so that patients can receive the care and support they need and deserve. The rare disease community struggles to have its stories told in mainstream media, which affects our ability to educate on rare disease awareness and fundraise, which can advance the rare disease community.

In many cases, rare disease patients have difficulty being correctly diagnosed, accessing appropriate care for their physical and mental health, and connecting with other rare/GNEM patients. Educating medical professionals on rare diseases such as GNEM could hopefully speed
up proper disease identification. For many, getting answers can take years of tests and improper diagnosis.

As with most non-profit organizations working in the rare disease sector, adequate financial support is lacking. Fundraising is challenging due to the limited grants available for rare diseases. With most of the community not directly affected or touched by a rare disease such as
GNEM, it can be hard to get people to care.

What are some of the pain points?
In addition to the above about what is lacking in the community, NDF is a small entity with limited resources. We only have two full-time employees and are careful to use our funds almost entirely for science and patient resources. While this aligns with our mission and work, it also limits what we can do.

Like many other organizations, NDF was greatly affected by the pandemic. We have been unable to hold our annual fundraiser for several years in a row now, which has been our most significant source of income. We must be creative to involve limited contractors, develop free programs, and lean on our small but mighty community of volunteers.

With that in mind, what NDF has been able to accomplish so far and provide to the GNEM community is remarkable.

What are your areas of expertise?
I am now a retired attorney and businessman with extensive management, fundraising, and non-profit development expertise. Throughout my career, I worked in London and Australia before coming to the US.

Please describe any major milestones your organization has hit or has coming up that you are proud of.
We are now paving the way to a treatment that we believe will halt the progression of GNEM in patients with our flagship plan, The International Gene Therapy Development Program (IGTDP), focused on gene therapy. NDF has assembled an international consortium of expert scientists, clinicians, and translational professionals to work together to reach the significant milestones of demonstrating “proof of concept” and holding a Pre-Investigational New Drug (pre-IND) meeting with the FDA. 

We are proud of our collaborative approach in this global endeavor and hope it will accelerate the long-awaited treatment for GNEM. The IGTDP is the culmination of more than a decade of NDF’s funding of international scientific research for GNEM towards treatment via grants which we would not have been able to do without the support of our donors.

We are dedicated and determined to make a real difference in the lives of our patients and support them the best we can as they wait for treatment. From our scientific advancements made through our International Gene Therapy Development Program (IGTDP) to the Multidisciplinary Clinic we offer to patients in the USA, our Emotional Wellness Clinic for our US and international patients, several patient support initiatives, including a monthly GNEM patient huddle and additional resources for care partners.  Our free speaker series is offered worldwide, including scientific presentations and patient-centric ones on adaptive technology, emotional wellness, diet and physical therapy, and more. These live sessions are accessible to the global community, and the recordings live in our learning library on our site.

Everything NDF does is possible thanks to our donors and sponsors who believe in supporting GNEM patients worldwide by contributing to our scientific research and patient programming.

What is your hope for the future for rare diseases?

I hope that in the not-too-distant future, there will be cures for many rare diseases, including GNEM.

In the meantime, as research advances, we will continue to find ways to help patients, their families, and caregivers improve their quality of life.

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