| Networking |
| Kathrin Gramsch |
TREAT-NMD Clinical Trials Coordination Centre: Efficiency of networking |
| Emma Heslop |
Engaging the Regulators to facilitate Antisense Oligonucleotide therapies for Duchenne Muscular Dystrophy |
| Rosaline Quinlivan |
Cochrane reviews: the best evidence for treating neuromuscular diseases? |
| Sarah Baumeister |
The EuroBioBank network: A vital link in translational research on neuromuscular disorders |
| Eduardo Tiazzano |
The GENAME project: an example of Spanish collaborative translational research in SMA |
| Sharon Hesterlee |
MDA Venture Philanthropy: A new approach to bridging the Valley of Death for Neuromuscular Diseases |
| Guenter Scheuerbrandt |
How to explain Duchenne research to Duchenne families |
| Jan Verschuuren |
ALADIN: All Against Duchenne In the Netherlands |
| Julaine Florence |
MDA Clinical Rsearch Network |
| Enrico Bertini |
TREAT-NMD and the ICC: A Fruitful Collaboration to Foster Clinical Trials and Research in SMA |
|
| General |
| Carlo Borsato |
Can Physical Activity or Iinflamation Influence The Outcome of LGMD2B? |
| Adrian Miller |
The genetic skeletal muscle channelopathies: Genotype-Phenotype correlation and longitudinal studies |
| Stefanie Bulst |
Divergent effects of pathogenic mutations in primary desminopathy on oligomer assembly and multimerization of desmin revealed by single particle spectroscopy |
| Natalia Garcia-Angarita |
Severe nemaline myopathy associated with consecutive mutations E74D and H75Y on a single ACTA1 allele |
| Francesco Conti |
Talin 1 and 2 are required for myoblast fusion, sarcomere assembly, and the maintenance of myotendinous junctions |
| Marina Dusl |
Limb-girdle congenital myasthenic syndrome with tubular aggregates - phenotypic clues for the entity |
| Slobodanka Todorovic |
Normal Phenotype in a person with Spinal Muscular Atrophy Genotype |
| Adrian Miller |
Development and Characterisation of an in vitro Model of Sporadic Inclusion Body Myositis (IBM) |
| Nicolas Deconinck |
A new ACTA1 mutation in two unrelated sporadic cases of neonatal form of CFTD: insights on the pathological mechanisms. |
| Marina Mora |
Clinical and molecular characterization of patients with defective ? - dystroglycan glycosylation. |
