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bis monoacylglycero aphosphate bmp as a circulating biomarker of lysosomal dysfunction in gne myopathy
Original title: “Bis(monoacylglycero)aphosphate (BMP) as a circulating biomarker of lysosomal dysfunction in GNE myopathy.”
Our team is currently finalizing ... gene therapy targeting GNE Myopathy and we anticipate that a Phase I clinical trial may begin within the next 12-months. Solve GNE remains focused on turning scientific progress into real momentum for GNE Myopathy patients and their families. Our commitment to funding additional research will help build a stronger path toward future treatment options for this rare disease... Our team is currently finalizing the IND application for the first gene therapy targeting GNE Myopathy and we anticipate that a Phase I clinical trial may begin within the next 12-months. Solve GNE remains focused on turning scientific progress into real momentum for GNE Myopathy patients and their families. Our commitment to funding additional research will help build a stronger path toward future treatment options for this rare disease. Solve GNE highlights new research, expanded funding efforts, and progress toward a Phase I clinical trial for GNE myopathy.... About Solve GNE Solve GNE is a nonprofit foundation dedicated to advancing research and treatment development for GNE myopathy. Through fundraising and scientific partnerships, Solve GNE works to accelerate progress toward therapies for patients and families affected by this rare muscle disease. LOS ANGELES, March 16, 2026 (GLOBE NEWSWIRE) -- Solve GNE, a nonprofit foundation dedicated to accelerating treatments for GNE myopathy, has announced major progress across its research, fundraising, and regulatory efforts. To date, the non-profit has raised more than $6.5 million in support of programs aimed at moving clinically viable therapies closer to approval.
Original title: “Solve GNE Highlights New Research, Expands Funding Efforts, and Advances Toward a Phase I Clinical Trial for GNE Myopathy”