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Today, the Committee for Medicinal Products for Human Use (CHMP) has returned a second negative…
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A study from MDUK-funded researchers at University College London (UCL) and Royal Holloway…
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Danish biotech company, Orphazyme, has assumed sponsorship of a phase 2/3 trial assessing the…
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US scientists have created a new mouse model of Duchenne muscular dystrophy (DMD) and treated it…
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AveXis is expanding its spinal muscular atrophy (SMA) clinical development programme for its gene…
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Lung function data from some of Sarepta’s Exondys 51 trials has been published in the Journal of…