Noelia Children's Foundation against congenital muscular dystrophy due to Collagen VI deficiency has received support…
Thanks to you, Fundación Noelia, achieves one of its goals for 2023 and donates $120,000 to Dr. Bönnemann’s team, from the National Institute of Neurological Disease and Stroke, in the USA, a project that establishes two gene therapies based on silencing and gene editing, very close to clinical trials for those affected by Collagen VI deficiency, as a consequence of more common and recurrent glycine substitutions.
This therapeutic approach aims to restore the collagen VI matrix so that the disease does not manifest itself.
Heartfelt thanks to all of you who, in one way or another, make research into this serious disease possible.
More info about the project:
DEVELOPING TWO RNA DIRECTED APPROACHES TO TREATING THE RECURRENT GLYCINE VARIANTS P.G284R AND P.G293R IN COL6A1