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Success Stories Award 2024 de la Swiss Biotech Association
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Maladies neuromusculaires
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Unstructured proteins as therapeutic targets for neuromuscular diseases
Open and reproducible pipeline for the acquisition and analysis of muscle MRI data in Facioscapulohumeral Muscular Dystrophy
Dissecting lysosomal signals to fight Pompe disease
Functional properties and epigenetic signature of quiescent and early activated human muscle reserve cells
Activation of human skeletal muscle stem cells:role of Orai3 ans its partner AHNAK2 in physiological condition and in Duchenne Muscular Dystrophy
Understanding the clinical spectrum associated with VMA21 deficiency
ANTXR2 as a key player in Collagen VI signaling in muscle stem cells: new therapeutic perspectives for COL6-related myopathies.
Molecular mechanisms of complement activation and neuromuscular disruption by combinations of autoantibodies from patients with Myasthenia Gravis
From the investigation of the role of SRSF1 in ALS/FTD to its targeting as a therapeutic strategy
Molecular crosstalk between muscles and motor neurons and its role in neuromuscular circuit formation
Molecular Diagnosis and Coping Mechanisms in Mitochondrial Myopathies
IPRIMYO: Immune-privileged, immortal, myogenic stem cells for gene therapy of Muscular Dystrophy
Effect of RYR1 mutations on muscle spindle function and their impact on the musculoskeletal system
Therapeutic potential of human myogenic reserve cells in Duchenne Muscular Dystrophy
Glutamine metabolism as a potential target for Duchenne Muscular Dystrophy
Targeting protein s-acylation during Tubular Aggregate Myopathy
Aggravating the phenotype of dystrophic mice for improving preclinical research and clinical translation for Duchenne muscular dystrophy
Characterization of autoreactive T cells in Guillain-Barré syndrome
A vascularized human muscle-on-a-chip to elucidate the contribution of endothelial-mesenchymal transition on the progression of muscular dystrophies
Characterization of a novel form of ALS associated with changes in the sphingolipid metabolism
Pre-clinical treatment of mouse models carrying recessive Ryr1 mutations with HDAC/DNA methyltransferase inhibitors.
New aspects of TGFβ signaling in muscle homeostasis and regeneration
Inhibition of sphingolipid synthesis as a treatment strategy for Duchenne muscular dystrophy
Tamoxifen in Duchenne muscular dystrophy (TAMDMD)
DNA aptamers against the DUX4 protein reveal novel therapeutic implications for FSHD
Facilitating diagnosis of critical illness myopathy using muscle excitability testing
Rapid Exploratory Imaging for High-resolution and Whole Extremity Coverage in MR Neurography
Deciphering novel mechanisms and effectors contributing to muscle dysfunction in Myotonic Dystrophy Type I
Can HDAC/DNA methyltransferase inhibitors improve muscle function in a congenital myopathy caused by recessive RYR1 mutations?
Identification of the critical regulators of protein synthesis and degradation in human muscle atrophy
Exploring peripheral B-cell-helper T cell phenotypes in the blood of patients with Myasthenia gravis using mass cytometry (CyTOF)
Molecular signature, metabolic profile and therapeutic potential of human myogenic reserve cells
A multicenter cross-sectional and longitudinal study of the Swiss cohort of Merosin-negative congenital muscular dystrophy
Targeting NADPH oxidase 4 in models of Duchenne muscular dystrophy
Characterizing the role of ER stress in C9orf72-linked ALS pathology
Inducing mitophagy with Urolithin A to restore mitochondrial and muscle function in muscular dystrophy
Motor unit action potentials analysis in patients with myopathies with a new wireless portable and multichannel Surface EMG device (WPM-SEMG)
Role and therapeutic potential of PLIN3 in neuromuscular diseases
Changes in ventilation distribution in children with neuromuscular disease using the insufflator/exsufflator technique: An observational study
Mechanism and function of genome organization in muscle development and integrity
Role and therapeutic potential of NADPH oxidases in a mouse model of Duchenne Muscular Dystrophy
Characterization of pathological pathways activated in muscles of patients with congenital myopathies with disturbed Ca2+ homeostasis
Creation of a study team to conduct an SMA 1-clinical trial at the Centre for Neuromuscular Diseases of the University Children's Hospital Basel (UKBB)
Novel treatment to stop progressive neuropathy and muscle weakness in multifocal motor neuropathy
Understanding the pathomechanisms leading to muscle alterations in Myotonic Dystrophy type I
Automated volumetry and quantitative MRI to diagnose peripheral nerve lesions – translational proposal for a new clinical diagnostic imaging tool
Novel approaches against Spinal Muscular Atrophy by targeting splicing regulators
Protective effects and mechanisms of action of tamoxifen in mice with severe muscular diseases
Role of the receptor FgfrL1 in the development of slow muscle fibers
Muscle velocity recovery cycles: A new tool for early diagnosis of critical illness myopathy
Generation of uncommitted human IPSC derived muscle stem cells for therapeutic applications
Transposable vectors for dystrophin-expression in a murine model for muscular dystrophy
Cardiac involvement in patients with Duchenne/Becker Muscular Dystrophy; an observational study
Deciphering the pathogenic mechanisms of C9ORF72 ALS
Enhancing estrogenic signalling to fight muscular dystrophies: Mechanisms of action and repurposing clinically approved drugs
Mechanisms and therapeutic potential of modulating PGC‐1α to alter neuromuscular junction morphology and function
Triggering human myoblast differentiation: from EGFR to myogenic transcription factors
Improving cellular therapies of muscle dystrophies by uncovering epigenetic and signaling pathways of muscle formation
Protein engineering in an attempt to increase the mechanical, integrin dependent cytoskeleton-matrix linkage in muscle fibers
Muscle velocity recovery cycles: a new tool for characterization of muscle disease in vivo
Excessive neurotrypsin activation and agrin cleavage-a pathogenic condition leading to sarcopenia-like muscle atrophy?
Evaluation of novel treatment strategies for dyspherlinopathies in mouse models
Cell therapy of LGMD2D by donor HLA-characterized human mesoangioblasts (hMABs) produced in GMP conditions
In search of small molecules targeting protein-RNA complex: a novel approach against Spinal Muscular Atrophy
Restoration of autophagy as a new strategy for the treatment of congenital muscular dystrophies
Development of magnetic resonance methods for functional imaging of the skeletal muscle
Targeting ER stress response: a potential mechanism for neuroprotection in Amyotrophic Lateral Sclerosis
Generation of uncommitted human IPSC derived muscle stem cells for therapeutic applications
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SEAL Therapeutics AG
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FSRMM
Chemin des Saules 4B
2013 Colombier
+41 78 629 63 92
philippe.rognon@fsrmm.ch
