This $2.5 Million Challenge Grant Could Fund GNE Myopathy Gene Therapy

According to a story from, the Neuromuscular Disease Foundation (NDF) recently announced that is has earned a $2.5 million challenge grant. This massive grant will go towards funding the development of a gene therapy for GNE myopathy, a form of muscular dystrophy that has become the focus of the NDF’s efforts. NDF is the largest nonprofit organization that is focused on supporting the GNE myopathy community.

About GNE Myopathy

GNE myopathy is a type of genetic disorder which is characterized by progressive muscle weakness that tends to appear in the early adult years of life. While generally rare, GNE myopathy appears to be more common in people of Iranian-Jewish descent. It is caused by a mutation of the GNE gene. Symptoms include weakness of the leg muscles in which, strangely, the quadriceps are spared. Other symptoms include weak index fingers, difficulty running and walking on the heels, progressively worsening coordination, and unusual characteristics upon muscle biopsy, such as rimmed vacuoles, inclusion bodies, and buildup of abnormal proteins. The heart may occasionally be affected. Treatment is mostly symptomatic and includes measures such as physical therapy. Most patients eventually require the use of a wheelchair. To learn more about GNE myopathy, click here.

What is a Challenge Grant?

A challenge grant is a unique style of fundraising in which a particular agreement is made between the recipient and the grant maker. This involves the recipient fundraising a certain amount of money in a specific, limited span of time. If this goal is achieved, then the grant maker will award the same amount to the recipient, therefore doubling their funds. This can be daunting task, but the potential rewards speak for themselves.

Gala for the Arts

To kick off its fundraising efforts, the NDF hosted a special Gala for the Arts event titled Past Forward- Through the Lens of Iranian Women. The event occurred November 11th, 2018, and will feature a number of Iranian female artists and musicians. This exciting event will start off the NDF’s new fundraising with a strong boost that will give them extra momentum.

To learn more about this event, click here.

To learn more about the NDF’s activities, click here.

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