Neuromuscular Disease Foundation Receives $2.5 Million Challenge Grant for HIBM Gene Therapy

Rare Daily Staff

The Neuromuscular Disease Foundation (NDF) was awarded a $2.5 million challenge grant from a Los Angeles-based private foundation to support NDF’s goal of developing a gene therapy for GNE myopathy, also known as HIBM.

GNE myopathy is a rare, genetic disease that atrophies the muscles of otherwise healthy young adults and may lead to physical debilitation within a decade of diagnosis. The disease affects people of all races, but is especially common in people of Persian, Jewish, Indian, European, and South East Asian descent. There is currently no treatment or cure for GNE Myopathy, although the National Institutes of Health is slated to enter its final phase of clinical trial for the drug ManNAc in 2019.

NDF will hold a Gala for the Arts on Sunday, November 11, at The Skirball Cultural Center in Los Angeles to help match the $2.5 million grant. The event will feature the work of more than 30 local Iranian-American artists and musicians, an auction, an award presentation, a dinner, and a concert featuring Sussan Deyhim and Chloe Pourmorady. All proceeds from the ticket sales, sponsorships and auction purchases will go to NDF’s initiatives.

“If we are to make the promise of gene therapy, the time and opportunity is now,” said NDF CEO Lale’ Welsh. “We have never been in a better place to benefit from the current technologies available in tandem with guidance from biotech partners, with whom we are currently in discussions, to help take this all the way to market.”

October 25, 2018
Photo: Lale’ Welsh, CEO Neuromuscular Disease Foundation