EurekAlert!

CHOP researchers develop first effective preclinical models for most common genetic cause of Leigh Syndrome

Translational findings in new models of SURF1 mitochondrial disease also suggest that two drugs, already FDA-approved for other conditions, may prevent neurological decompensation in Leigh syndrome ...
PharmiWeb

First Clinical Data on Ornithine Transcarbamylase Deficiency Program to be presented at the European Society of Gene & Cell Therapy 31st Annual Meeting 2024

London, UK, 01 October 2024 – Bloomsbury Genetic Therapies Limited (“Bloomsbury”), a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare ...
Medindia

First Effective Preclinical Models for Most Common Genetic Cause of Leigh Syndrome

SURF1 deficiency is the most common nuclear genetic cause of Leigh syndrome. The SURF1 gene is critical for transforming nutrients into energy that can be used by our cells. Disease-causing mutations ...