• Die Fortschritte der Forschung sind die einzige Hoffnung der Patienten auf wirksame Therapien!

  • Unterstützen Sie die Forschung an seltenen genetischen Muskelkrankheiten!

International scientific congress on Spinal Muscular Atrophy

Krakow, Poland, 25-27 January 2018

The best experts from all five continents are expected to present and compare their latest work and discoveries not only in basic, but also in clinical research and in therapies.

Infos and registration


ENMC Workshopsbericht

Clinical trial readiness for Calpainopathies. 17-19 September 2017

Auf deutsch verfügbar

Recommendations for treatment of Mitochondrial DNA maintenance disorders. 16-18 June 2017



TREAT-NMD International Conference

27-29 November 2017, Freiburg (Germany)

The aim of this international conference is to share progress and lessons learned in the area of translational medicine in inherited neuromuscular diseases and plan for the delivery of future therapies to patients.



Re(Act) Congress

7-10 March 2018, Bologna, Italy

The congress aims to bring together world leaders and young scientist from a variety of breaking through scientific field to present cutting edge research, to discuss results and to exchange ideas. Moreover, many patients and patient organization, which are committed in research, will be present to share their experience.

Abstract submission until 15 December 


An unbiased silencing screen in muscle cells identifies miR-320a, miR-150, miR-196b, and miR-34c as regulators of skeletal muscle mitochondrial metabolism.

Dahlmans D, Houzelle A, Andreux P, Jörgensen JA, Wang X, de Windt LJ, Schrauwen P, Auwerx J, Hoeks J.
Mol Metab. 2017 Nov;6(11):1429-1442.

14 November 2017

Split GFP technologies to structurally characterize and quantify functional biomolecular interactions of FTD-related proteins.

Foglieni C, Papin S, Salvadè A, Afroz T, Pinton S, Pedrioli G, Ulrich G, Polymenidou M, Paganetti P.
Sci Rep. 2017 Oct 25;7(1):14013.

07 November 2017

PGC-1α affects aging-related changes in muscle and motor function by modulating specific exercise-mediated changes in old mice.

Gill JF, Santos G, Schnyder S, Handschin C.
Aging Cell. 2017 Oct 25.

25 Oktober 2017

Tandem hnRNP A1 RNA recognition motifs act in concert to repress the splicing of survival motor neuron exon 7.

Beusch I, Barraud P, Moursy A, Cléry A, Allain FH.
Elife. 2017 Jun 26;6.

20 Oktober 2017