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spinal muscular atrophy (SMA)

Healthcare solutions provider, Novartis, is set to gain further ground in the area of gene therapy through its agreement to acquire AveXis — US-based clinical stage gene therapy company — for $8.7 billion. more

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Biotech company Scholar Rock has closed a $47 million Series C financing round, which will be used to advance its innovative pipeline for spinal muscular atrophy (SMA), immune-oncology, fibrosis and iron-restricted anaemias. more

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Research from the University of Warwick has shown that most people are in favour for newborn screening of the potentially fatal condition spinal muscular atrophy (SMA). more

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The CHMP has given a positive opinion recommending that Biogen’s treatment for patients with spinal muscular atrophy (SMA), SPINRAZA (nusinersen), be granted marketing authorisation. more

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