Myotubular Myopathy (MTM or MM)

MTM: Down With Dynamin 2

A French research team has found that reducing levels of a protein called dynamin 2 has potential as a strategy to treat myotubular myopathy (MTM), a form of centronuclear myopathy (CNM), and that it could have implications for other nerve and muscle disorders as well.

New Forms of Genetic Testing Improve Diagnosis, Raise Questions

“Knowing, if not all, is almost all,” said Matthew Harms, a neurologist and neurophysiologist from Washington University in St. Louis, in his presentation on genetic testing for neuromuscular disorders at the 2014 MDA Clinical Conference, held in Chicago March 16-19.

MTM Natural History Study Seeks Participants

Update (May 20, 2014): This story has been updated to reflect additional information received May 20, 2014, from Michelle Nelken at Valerion Therapeutics, including the joint sponsorship of this study by Valerion and Genethon.

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Animal Studies Bode Well for MTM Gene Therapy

CNM/MTM Registry, World Map Seek Participants

A patient registry and world map of people with centronuclear myopathies (CNM), including myotubular myopathy (MTM), are being developed and are seeking participation from people with these disorders or their family members.

Titin Mutations Can Cause CNM

Researchers in the United States and France, supported in part by MDA, have established that mutations in the titin gene are a cause of centronuclear myopathy (CNM), a group of muscle disorders characterized by variable degrees of weakness and cell nuclei that are abnormally located toward the center of muscle fibers rather than around the perimeter.

MTM — Valerion Therapeutics

The Muscular Dystrophy Association has awarded $1,195,762 over two years to biotechnology company Valerion Therapeutics (formerly 4s3 Bioscience) for development of a treatment for myotubular myopathy (MTM).

MTM: MDA Funds Development of Myotubularin-Based Treatment

The Muscular Dystrophy Association has awarded $1,195,762 over two years to biotechnology company Valerion Therapeutics (formerly 4s3 Bioscience) for development of a treatment for myotubular myopathy (MTM).

Moving Toward 'Next-Generation' Gene Therapy

Planning for the next generation of gene and stem cell therapies for muscular dystrophies — even as the first generation is still under development — was the theme of a joint symposium sponsored by MDA and the Association Française Contre les Myopathies (French Association Against Myopathies, or AFM) at the 16th annual meeting of the American Society of Gene & Cell Therapy (ASGCT).

MTM Phone-Based 'Event' Study Now Open

The Myotubular Myopathy Event Study, a telephone-based survey, will gather information about MTM-associated events, such as emergency room visits, hospitalizations, medication reactions, and complications from medical procedures, as well as improved or declined motor and respiratory function.

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