In a finding with implications for one of the most promising cutting-edge medical treatments, researchers evaluated several ...

The upside in Sarepta's share price can be attributed to the encouraging sales performance of its DMD gene therapy, which has demonstrated blockbuster potential.

Microbial neutralization must constantly achieve a six-log reduction threshold of Geobacillus stearothermophilus and comply ...

Sidra Medicine, a member of Qatar Foundation, has made significant strides in improving patient care and healthcare access in ...

One-year-old Celine has an incurable form of muscular dystrophy and will receive her first round of stem cell therapy after ...

Thirty-one members of the University of Chicago faculty have received distinguished service professorships or named ...

Vebeglogene autotemcel is under clinical development by Lantu Biopharma and currently in Phase II for Beta Thalassaemia.

New research uncovers why certain individuals with disease-causing genes remain symptom-free. Researchers at Columbia ...

This study describes a valuable new model for in vivo manipulation of microglia, exploring how mutations in the Adar1 gene within microglia contribute to Aicardi-Goutières Syndome. The methodology is ...

A Look Back at an Exciting 2024 LONDON, UNITED KINGDOM / ACCESSWIRE / January 6, 2025 / 2024 has been an incredible year for Genflow as we advance our mission to develop novel therapeutics that ...

New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety. Updated ...