Other Myopathies
Researchers conducting a study of medical changes and complications in myotubular myopathy (MTM) are seeking individuals with MTM or their parents for 12 monthly phone interviews
Drug testing for Duchenne MD, Leigh syndrome and Pompe disease moves forward; and two MDA-supported mouse studies suggest leads for myasthenia gravis and periodic paralysis
Disease registry seeks input from people with congenital muscular dystrophies, congenital myasthenic syndromes or other congenital muscle disorders, including those with no clear diagnosis
Mice with a disorder mimicking myotubular myopathy showed improvements in muscle strength and function in response to a myotubularin-based enzyme replacement therapy
In the past, children with rapidly progressive neuromuscular scoliosis often underwent growth-stopping spinal fusion. Today, there are more options.
MDA-supported scientists have developed a research mouse with a calcium channel mutation and a disorder that mimics human hypokalemic periodic paralysis
A competition to identify and explain genetic mutations using genome sequencing has solved an 11-year mystery for the family of a boy with centronuclear myopathy
Researchers are studying the 'natural history' of centronuclear/myotubular myopathies, central core disease and multiminicore disease, with an eye to future trials
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