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 ...
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 ...
WAKEFIELD, Mass.--(BUSINESS WIRE)--Myrtelle Inc. (“Myrtelle” or the “Company”), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, ...
(HealthDay News) – In children with Canavan disease, a hereditary leukodystrophy that results in the degeneration of brain white matter caused by mutations in the aspartoacylase (ASPA) gene, gene ...
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 ...
BROOKLYN, New York City -- A family in Brooklyn is on a mission to save their children, who are suffering from a rare genetic disorder. They're trying to raise money for a treatment they say could ...
- These results are the first reported demonstration of rapid and robust treatment changes in key disease markers associated with the severity of disease - Initial pharmacodynamic results for two ...
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 ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Myrtelle Inc. announced updated findings of the ...
CONCORD, Calif. -- Fraternal twins Yael and Yoel Ilinetsky in Concord, California, are battling an extremely rare genetic disease called Canavan Disease. They're lacking an enzyme needed to break down ...
BROOKLYN, New York City -- A family in Brooklyn is on a mission to save their children, who are suffering from a rare genetic disorder. They're trying to raise money for a treatment they say could ...
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