Projects

The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. A network-biology based approach for the development of SMN-independent treatments

    Principal investigator(s) :
    Prof. Dr. Peter Claus
    Institution :
    Medizinische Hochschule Hannover, Germany
    Grant :
    €145,900
    Grant Type:
    Operating Grant
    Call number :
    10
    Start year :
    2020
    Duration :
    2 years
    Status :
    Not yet started


    Professor Peter ClausProfessor Peter Claus, of the Department of Neuroanatomy and Cell Biology at Hannover Medical School, in Hannover, Germany, was awarded an SMA Europe grant to develop a network-biology based approach to detect and test SMN-independent treatment options.

    Background

    SMA is caused by defects in a single gene leading to low levels of one protein, SMN. There is now an approved treatment, Spinraza, which restores levels of this protein. However, not all patients benefit from this drug and since it already elevates SMN-levels, other therapeutic strategies are needed.

    What is Professor Claus proposing to do and why?

    Professor Claus will investigate how to contribute to such SMN-independent approaches. Together with his team, he has detected many changes in mice which develop SMA and used computer-simulations to see how these changes associate in a network. Highly connected changes are predicted to be more important for disease progression than others. Professor Claus will normalise these changes and test whether this has therapeutic advantages.