Therapeutic advances in spinal muscular atrophy
Spinal muscular atrophy (SMA) is a rare neuromuscular condition, characterized by loss of motor neurons as a result of a mutation in the survival motor neuron gene. This results in muscle wasting and in the most common and severe type, death before 24 months. Over the recent years there has been a dynamic shift in the therapeutic options for these patients involving both approved therapies, including gene therapy, and access to clinical trials in genetic modifying therapies to indirectly improve the survival motor neuron protein level and hence strength, muscle promotor therapies, up/down regulation of modifier genes and SMN independent therapies.
Source: Paediatrics and Child Health - Category: Pediatrics Authors: Tracey Anne Willis Tags: Occasional review Source Type: research
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