Rare Disease Foundation takes on 2.5 Million challenge grant to fund Gene Therapy

Thursday, October 25, 2018 Genetics & Stem Cells News
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The Neuromuscular Disease Foundation is awarded a 2.5 Million matching grant from a private foundation towards gene therapy and its necessary studies as a potential cure for rare muscle disease, GNE Myopathy (also known as HIBM).

BEVERLY HILLS, Calif., Oct. 24, 2018 /PRNewswire-PRWeb/ -- The Neuromuscular Disease Foundation (NDF) -the world's leading

nonprofit organization focused on a rare form of muscular dystrophy known both as GNE Myopathy (GNEM) and HIBM- has achieved a near-impossible goal in the world of rare diseases: NDF has achieved a $2.5 million challenge grant from a local private Foundation to support NDF's goal of developing gene therapy for GNE Myopathy.

"We are beyond grateful to have been selected for this grant. We need all GNEM stakeholders and community members to rally behind this urgent cause by raising the $2.5 million so we can obtain the matching gift of $2.5 million," said NDF Chief Executive Officer, Lale' Welsh. "If we are to make the promise of gene therapy, the time and opportunity is now. 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."

In order to help meet its lofty mission of matching the $2.5 million, NDF is hosting a Gala for the Arts on Sunday, November 11 at The Skirball Cultural Center in Los Angeles featuring the work of over 30 Iranian-American artists and musicians. The event will feature an art show of local, emerging and prominent artists, 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.

David Dahl who assisted Lale' Welsh in obtaining the grant added: "The reason the Foundation chose NDF to receive this award was because of their commitment to data sharing, their strategy for gene therapy to work with biopharma in order to see it through to fruition, which in turn will help other rare muscle diseases scale in the same way."

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. There is currently no treatment or cure for GNEM, although the National Institutes of Health (NIH) is slated to enter its final phase of clinical trial for the drug ManNAc in 2019. While this disease affects people of all races, it is especially common in people of Persian, Jewish, Indian, European, and South East Asian descent.

NDF is dedicated to enhancing the quality of life for people living with GNE Myopathy by funding clinical research through collaborations with scientists and physicians worldwide, and through programs related to outreach, education and prevention for affected individuals and their families and caregivers.

 

SOURCE Neuromuscular Disease Foundation



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