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Spinal Muscular Atrophy (SMA) beyond motoneuron degeneration: multi-system aspects

Project description

Targeting multi-organ defects for the treatment of spinal muscular atrophy

Spinal muscular atrophy (SMA) is a motor neuron disease caused by reduced levels of the survival of motor neuron (SMN) protein. The SMN protein is ubiquitously expressed in the central nervous system (CNS) and peripheral organs. Restoring SMN protein levels in the CNS constitutes a potent strategy that has led to the approval of therapies for clinical use. The aim of the EU-funded SMABEYOND project is to investigate the role of the SMN protein in peripheral organs and determine the intrinsic defects encountered in SMA patients. The ultimate goal is to develop SMA patient-derived disease models and test the therapeutic effect on peripheral organs of systemic SMA therapy.

Call for proposal

H2020-MSCA-ITN-2020
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Funding Scheme

MSCA-ITN-ETN - European Training Networks

Coordinator

MEDIZINISCHE HOCHSCHULE HANNOVER
Address
Carl-neuberg-strasse 1
30625 Hannover
Germany
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 252 788,40

Participants (7)

UNIVERSITAIR MEDISCH CENTRUM UTRECHT
Netherlands
EU contribution
€ 265 619,88
Address
Heidelberglaan 100
3584 CX Utrecht
Activity type
Higher or Secondary Education Establishments
STIFTUNG TIERAERZTLICHE HOCHSCHULE HANNOVER
Germany
EU contribution
€ 252 788,40
Address
Bunteweg 2
30559 Hannover
Activity type
Higher or Secondary Education Establishments
CONSIGLIO NAZIONALE DELLE RICERCHE
Italy
EU contribution
€ 261 499,68
Address
Piazzale Aldo Moro 7
00185 Roma
Activity type
Research Organisations
KLINIKUM DER UNIVERSITAET ZU KOELN
Germany
EU contribution
€ 252 788,40
Address
Kerpener Strasse 62
50937 Koeln
Activity type
Higher or Secondary Education Establishments
UNIVERSITY COLLEGE LONDON
United Kingdom
EU contribution
€ 303 172,56
Address
Gower Street
WC1E 6BT London
Activity type
Higher or Secondary Education Establishments
THE UNIVERSITY OF EDINBURGH
United Kingdom
EU contribution
€ 303 172,56
Address
Old College, South Bridge
EH8 9YL Edinburgh
Activity type
Higher or Secondary Education Establishments
FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA
Spain
EU contribution
€ 250 904,88
Address
Passeig Vall D Hebron 119-129 Edificio De Recerca
08035 Barcelona
Activity type
Research Organisations