Credit: Shutterstock. The gene therapy candidate is designed to deliver the ASPA gene directly to the oligodendrocyte cells in the fluid surrounding the brain and spinal cord. The Food and Drug ...
Groundbreaking study demonstrates that targeted oligodendrocyte gene therapy leads to decreased N-acetylaspartate (NAA) levels and increased brain myelin volume, resulting in promising functional ...
Investigators have settled a long-standing controversy surrounding the molecular basis of an inherited disorder that historically affected Ashkenazi Jews from Eastern Europe but now also arises in ...
Analyses of the first three patients at 6 months post-treatment showed increases in white matter and myelin and improvements on validated functional scales To date, 5 patients have been treated in the ...
NEW YORK (CBSNewYork)-- A family from Brooklyn is trying to raise more than $2 million to help their sons, who have a rare genetic disease. It's a struggle countless families face, especially when ...
When Jennie Landsman’s son Benny was 6 months old, she noticed he struggled to sit up, roll over or scoot like other babies. Worried, she took him to a neurologist. “I started to get a little bit ...
WAKEFIELD, Mass.--(BUSINESS WIRE)--Myrtelle Inc. (“Myrtelle” or the “Company”), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, ...
Dominic Gessler lifts two squirming mice from their cages. Sherri Epstein flinches, but Gessler coaxes her to hold out her hands. He places the small animals in her palms, and they skitter in tiny ...
NEW YORK, Sept. 16, 2025 /PRNewswire/ -- Myrtelle Inc. ("Myrtelle" or the "Company), a pioneering clinical-stage gene therapy company developing transformative treatments for neurodegenerative ...
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