Background: TREAT-NMD is an international network of excellence facilitating collaborative research in neuromuscular disease (NMD). Having developed a range of infrastructures, the network aims to […]
Objective: Venous thromboembolism (VTE) is a well-established cause of significant morbidity and mortality in the adult population with risk that increases with higher age and […]
Centronuclear myopathies (CNMs) are a group of conditions characterized by skeletal muscle weakness and centrally located skeletal fiber nuclei on muscle biopsy. Onset of muscle […]
Background Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease caused by reduced levels of survival of motor neuron (SMN) protein due to deletions […]
Background Individuals with spinal muscular atrophy (SMA) may have atypical weight with possible correlation to other disease morbidity, such as feeding difficulties and reduced mobility. […]
Objective: The primary purpose of SMART (NCT04851873), an ongoing, open-label, single-arm, multicenter study, is to systematically collect safety/tolerability data for onasemnogene abeparvovec in symptomatic spinal […]
Background Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease caused by reduced levels of survival of motor neuron (SMN) protein due to deletions […]
Objective: SPR1NT (NCT03505099) was a multicenter, open-label, Phase III study to evaluate the safety and efficacy of onasemnogene abeparvovec in presymptomatic patients with spinal muscular […]
Background Spinal muscular atrophy (SMA) is a genetic, neuromuscular disease characterized by progressive motor neuron degeneration. With the recent availability of disease-modifying therapies (DMTs) and […]