Funded Research Projects

2022

Are vascular defects important in SMA?

Principal investigator(s):

Professor Simon Parson

2022

Investigating microvasculopathy in SMA patients and their response to nusinersen, onasemnogene abeparvovec and risdiplam.

Principal investigator(s):

Dr. Haiyan Zhou

2022

Uncovering the mechanism of action and synergistic potential of an SMA repositioned therapy

Principal investigator(s):

Professor Marina Boido

2020

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

Principal investigator(s):

Prof. Dr. Peter Claus

2020

Molecular mechanisms of synaptic transmission of the sensory motor-circuit in spinal muscular atrophy

Principal investigator(s):

Dr. Christian Simon

2020

SMA: Mechanisms implicated in the perturbation of the calcium homeostasis and essay of a new therapeutic molecular target

Principal investigator(s):

Professor Lucia Tabares

2020

Unravelling the role of Stathmin-2 in SMA as a key a key cause of motor neuron vulnerability and as a therapeutic target

Principal investigator(s):

Dr. Monica Nizzardo

2017

Combinatorial ASO therapy using SMN-dependent and SMN-independent protection against SMA

Principal investigator(s):

Dr. Laura Torres-Benito