Neuromuscular Disorders (NMD) cover a group of devastating congenital conditions which can affect the whole body which include spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS, motor neurone disease) and Duchenne muscular dystrophy (DMD). While muscle weakness causes minor problems such as twitching, cramps and aches and pains, more serious consequences are mobility problems, reduced heart function and breathing ability. Most NMD have no cure so the goal of treatment has been to improve symptoms, increase mobility and lengthen life. NMD are an active field of research and recent and ongoing clinical trial data offer real hope for new treatments to extend current treatment goals.
The SMA Learning Zone contains easily absorbed and concise information about this devastating congenital neuromuscular disease. It will teach you about SMA as a whole-body disease, what role the SMN1/2 genes play and how targeting the SMN2 gene can have therapeutic benefit by producing functional SMN protein.
Learn how to recognise SMA with discussions of the epidemiology, the disease symptoms, the differences between SMA types I-IV and the burden of disease.
Increase your understanding of SMA treatment about current disease management, guidelines or recently concluded and ongoing clinical trials and how these will change the future landscape of SMA therapy.
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