Patient and Parent Perspectives on the Use of Nusinersen for Spinal Muscular Atrophy
At the end of 2016, the U.S. Food and Drug Administration (FDA) approved nusinersen for the treatment of spinal muscular atrophy (SMA).1 Because of the high threshold for FDA approval, an abundance of data on nusinersen and its physiological effects on SMA patients...
Nusinersen in Spinal Muscular Atrophy: What is Still Unclear
In 2016, nusinersen, or Spinraza, became the first drug for spinal muscular atrophy (SMA) ever approved by the U.S. Food and Drug Administration.1 The drug acts to combat low survival motor neuron (SMN) protein in the central nervous system by correcting the splicing...
Spinal Muscular Atrophy and the Role of MicroRNA
Mutations in the survival motor neuron 1 gene (SMN1), which result in reduced expression and lower levels of the full-length SMN protein, are responsible for spinal muscular atrophy (SMA).1 Given the role that microRNAs (miRNAs) play in gene expression, it is perhaps...Disease Modifying Therapies in Spinal Muscular Atrophy: Preclinical Products
The therapies that are being developed for spinal muscular atrophy (SMA) generally fall into two categories: those that aim to increase levels of survival motor neuron (SMN) protein and those that are SMN-independent.1 The therapies that aim to increase SMN protein...
Pain Management in Spinal Muscular Atrophy
Increased pain is significantly associated with lower levels of health, social function, and vitality.1 The results of studies on animal models of neuropathic pain suggest that the abnormal excitability of dorsal horn neurons in spinal muscular atrophy (SMA) may lead...HCP — SMA News Today
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