Novel treatment of Neuromuscular Disease
This translational research program mentored by NMD Pharma in Aarhus and the Membrane Protein Structural Biology Group at University of Copenhagen aims at improving the life of people suffering from neuromuscular diseases. The ambition is to develop novel therapies that restore muscles ability to capture signals from the central nervous system. This ability is gradually lost in neuromuscular diseases such as the incurable ALS, which Stephen Hawkings suffered from.
The research program is based on recent major breakthroughs in the two mentor groups: NMD Pharma has discovered that inhibition of certain proteins reinstate muscle function, and the University based research team has experience with elucidating detailed structures (atomic models) of these proteins. The approach will be to optimize NMD Pharma inhibitors using e.g. a technique that recently was awarded the Nobel Prize, cryo-electron microscopy, and through evaluation of improved lead compounds at a cellular, organ and living animal level.
Basic Mentor: Associate Professor Pontus Gourdon, Department of Biomedical Sciences (BMI), University of Copenhagen
Clinical Mentor: Dr. Claus Elborg Olesen, Co-founder and CBO, NMD Pharma