| Methodology and Characterisation |
| Elizabeth Greally |
Magnetic resonance imaging of cardiac function in mouse models for muscular dystrophy associated cardiomyopathy |
| Tina Duong |
Mild exercise improves skeletal muscle performance in a mouse model of limb girdle muscular dystrophy 2B |
| Raffaella Willmann |
TREAT-NMD- Activity 07: Accelerate preclinical phase of new therapeutic treatment development |
| Shinya Kamauchi |
Urinary level of prostaglandin D2 metabolites in animal models of Duchenne muscular dystrophy |
| Marina Mora |
An in vitro cell model to further elucidate mechanisms of muscle fibrosis and test possible anti-fibrotic agents |
| Bassem F. El-Khodor |
Phenotyping Neonatal models of neuromuscular degeneration as a prelude for drug candidate evaluation. |
| Miranda Grounds |
The immediate consequences of treadmill exercise on adult dystrophic mdx mice |
| Kanneboyina Nagaraju |
Preclinical Drug Trial Efforts for Muscular Dystrophy: Methods and End points |
| Maaike van Putten |
Effect of a mild exercise regime on disease parameters in the mdx mouse model |
| Sabine De La Porte |
MALDI reveals membrane lipid profile reversion in MDX mice |
| Joe N. Kornegay |
Muscle Hypertrophy and Contractures in a Myostatin Heterozygote Null GRMD Dog |
| Richard Lovering |
Structural and functional evaluation of branched myofibers in mdx mice |
| Rebecca Pruss |
Modelling of SMA in mice - novelties and limits |
| David Bendahan |
Non invasive Assessment of skeletal muscle function in mouse using 1H-MR imaging and 31P-MR spectroscopy |
|
| Antisense approaches in NMD |
| Peter Sazani |
AVI-5038: Initial Efficacy and Safety Evaluation in Cynomolgus Monkeys |
| Annemieke Aartsma-Rus |
Applicability of Antisense-Mediated EXON Skipping for Duchenne Muscular Dystrophy Mutations |
| Peter 't'Hoen |
Dual exon skipping in myostatin and dystrophin as a potential therapy for Duchenne muscular dystrophy |
| Pietro Spitali |
Guidelines for exon skipping quantification in the DMD gene |
| Annemieke Aartsma-Rus |
Long Term Systemic Antisense-Mediated Exon Skipping in Dystrophic Mouse Models |
| Linda Popplewell |
Multiexon skipping in Duchenne muscular dystrophy |
| Alberto Malerba |
Chronic long term administration of low and high doses of phosphorodiamidate morpholino oligomer ameliorates the dystrophin phenotype in mdx mice |
| Dr Matthew JA Wood |
Muscle and Heart Targeted Splice Correction for DMD |
| Adrian Krainer |
Antisense Correction of SMN2 Splicing in the CNS of Mouse Models of Spinal Muscular Atrophy |
| Peter Sazani |
Repeat Dose Mechanistic Toxicology Evaluation of AVI-4225 PMO in mdx mice |
| Peter Sazani |
Repeat Dose Toxicology Evaluation of AVI-4658 PMO in mdx mice |
| Peter Sazani |
Repeat Dose Toxicology Evaluation of AVI-4658 PMO in Cynomolgus Monkeys |
| Sue Fletcher and Steve Wilton |
Exon Skipping for Non-Deletion Dystrophin Mutations |
| Sue Fletcher |
Induced non-productive splicing to study Muscle Gene Expression |
| Takashi Saito |
Analysis of exons 6 and 8 skipping in cells from an exon-7 deleted DMD patient: direct application of antisense sequences found in study with canine muscular dystrophy. |
|
| Cell and gene therapy approaches |
| Stefanie Schinkel |
Efficient systemic AAV9-mediated microdystrophin gene transfer in the mdx heart |
| Severine Pouillot |
Automation of biodistribution study |
| Sabrina Sacconi |
Cell Therapy for Muscular Dystrophy: CD34 Negative Muscle Derived Cells present High Myogenic and No Adipogenic Potential |
| Yvan Torrente |
Combining Stem Cells and Exon Skipping Aapproach to Treat Muscular Dystrophies |
| Vincenzo Nigro |
Gene therapy and glucocorticoids for muscular dystrophy and cardiomyopathy of BIO14.6 hamster |
| Seng H. Cheng |
Gene Therapy or Antisense Oligonucleotide-mediated Augmentation of SMN Levels for Spinal Muscular Atrophy. |
|
| Therapeutic Targets |
| Li Niu |
Developing AMPA Receptor Aptamers as New Drug Candidates for ALS |
| Mahasweta Girgenrath |
Up-regulation of TGF beta signaling in MDC1A |
| Jennifer Lachey |
Activin Receptor Type IIB Inhibition Improves Strength and Function of Dystrophic Muscle |
| Kosuke Aritake |
Chemotherapy of Duchenne muscular dystrophy |
| Paolo Bonaldo |
Genetic and pharmacological inhibition of cyclophilin D rescues mitochondrial defects and prevents muscle apoptosis in collagen VI myopathic mice |
| Annamaria De Luca |
Exercised dystrophic mdx mice and specific modulation of disease-sensitive indices by drugs clinically relevant for duchenne muscular dystrophy: effects of a chronic treatment with the angiotensin-converting enzyme inhibitor enalapril |
| |
|
| |
|
