nusinersen
National Institutes of Health
Papers overview
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In August 2013, the Centers for Medicare and Medicaid Services (CMS) Open Payments Program (OPP) made eligible payment…
at younger than 1 year of age in Japan. The present patient had no history of honey intake, or of corn syrup or vegetable juice…
Background Spinal muscular atrophy (SMA) Type 1 is a neuromuscular disorder that has traditionally resulted in death from…
Nusinersen is the first approved drug to treat spinal muscular atrophy (SMA). Its periodic intrathecal delivery may cause…
Spinal muscular atrophy (SMA) is the most common genetic cause of infant mortality. SMA is a spectral disorder and is categorised…
Objective: To examine distance walked and fatigue during the six minute walk test (6MWT) in nusinersen-treated children with…
KEY POINTS
Canada is one of the few developed countries without a regulatory framework for orphan drugs.[1][1] Orphan drugs are…
Background Classic proximal 5q-SMA is a progressive motor neuron disorder leading to muscle weakness and atrophy and is the most…