How is focal muscular atrophy (FMA) treated?

Updated: Feb 20, 2018
  • Author: Sridharan Ramaratnam, MD, MBBS; Chief Editor: Helmi L Lutsep, MD  more...
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Treatment of focal muscular atrophy (FMA) varies according to the cause. The common causes (eg, monomelic amyotrophy, PPMA, SMA) have no specific treatment.

When patients with these conditions have disability, the treatment consists of physical and occupational therapy and rehabilitation.

A report of increase in the SMN2 messenger RNA levels in vivo among 7 of 13 patients with spinal muscular atrophy treated with valproic acid raises possibilities of in vivo activation of causative genes in inherited diseases. [55]

Some of the therapeutic strategies that have been tested in SBMA, fall into four main categories: (i) gene silencing; (ii) protein quality control and/or increased protein degradation; (iii) androgen deprivation therapies using leuprorelin and dutasteride; and (iv) modulation of androgen receptor function. Various therapeutic strategies have been effective in transgenic animal models, and research is ongoing to translate these strategies into safe and effective treatment in humans. [56]

An open trial of clenbuterol among patients with spinal and bulbar muscular atrophy (SBMA) found significant and sustained increase in walking distance covered in 6 minutes and forced vital capacity between the baseline and the 12-month assessments (P< .001), suggesting class IV evidence that clenbuterol may be effective in improving motor function. [57]  A randomized trial found no significant effect of dutasteride on the progression of muscle weakness in SBMA. [58]

Counsel patients concerning the benign nature of the illness once the diagnosis is confirmed.

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