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Dynacure announces publication of Hierarchical Bayesian modelling of disease progression in Orphanet Journal of Rare Diseases
Bayesian statistical approach based on natural history study could address clinical trial design challenges in centronuclear myopathy
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STRASBOURG, France and PHILADELPHIA, Jan. 15, 2021 /PRNewswire/  Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, announced the publication of a paper entitled,
Hierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy (CNM). The paper was published this month in Orphanet Journal of Rare Diseases, a scientific, peer-reviewed publication and can be accessed here. ....

United States , France General , Laurent Servais , Centronuclear Myopathies , Neuromuscul Disord , Brad Carlin , Institute Of Myology , Oxford Neuromuscular Center , University Of Oxford , Tekla Capital Management , European Union , Pharmalex Group , Ionis Pharmaceuticals , Department Of Pediatrics , Hierarchical Bayesian , Orphanet Journal , Rare Diseases , Pediatric Neuromuscular Diseases , Neuromuscular Center , Senior Advisor , Data Science , Leen Thielemans , Chief Development Officer , Ionis Pharmaceutical , Orphan Drug , Hereditary Spastic Paraplegias ,