Isis Pharmaceuticals is launching a phase 1 trial of ISIS-DMPKRx, an experimental drug for type 1 myotonic muscular dystrophy
Update (June 11, 2014): This story has been updated with additional information about the phase 1 trial of ISIS-DMPKRx in healthy volunteers and the planned trial in people with type 1 MMD, as well as the availability of a fact sheet on this drug provided by Isis Pharmaceuticals...
Researchers at the University of Michigan are conducting an anonymous, online survey to probe patients' impressions of the impact of their neuromuscular conditions
Update May 21, 2014: According to investigator Sindhu Ramchandren, 922 people had responded to this survey as of May 19, 2014. The study was closed to new participants at 5 p.m. EDT that day. At the investigator's request, the link to the online survey has been removed. Results will be announced when they become available...
This second of a series of three stories covering the 2014 MDA Clinical Conference discusses pain in neuromuscular disorders
The 2014 MDA Clinical Conference, held in Chicago March 16-19, was attended by some 500 people, mostly physicians and other health care professionals.
This first of a series of three stories covering the 2014 MDA Clinical Conference discusses implications of new types of genetic testing
“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.
MDA's 2014 Clinical Conference on neuromuscular disease brings together health professionals from across the country and focuses on improving lives through excellence in care
"Optimizing Care: Improving Lives Through Clinical Excellence" is the theme of the 2014 MDA Clinical Conference, which takes place March 16-19 at the Hyatt Regency in Chicago. A secondary theme — "I am progress" — also will be in evidence, referencing the crucial role that MDA clinic team members nationwide have in providing expert medical care in neuromuscular disease and identifying new...
High energy, optimism prevailed at a February 2014 congressional briefing for Capitol Hill staffers on reauthorizing key muscular dystrophy legislation
"The energy in the room was high, and the mood was optimistic," said Annie Kennedy, MDA's senior vice president of advocacy, speaking about the Congressional briefing on reauthorization of the MD-CARE Act that she attended and helped to organize. The briefing was co-hosted by MDA and other muscular dystrophy organizations in Washington, D.C., on Feb.
MDA-supported researchers have developed compounds that block harmful interactions between a needed protein and abnormal RNA in cells with a type 2 myotonic dystrophy defect
MDA-supported investigators have designed two new compounds that precisely target the molecular defect that underlies type 2 myotonic muscular dystrophy (MMD2, or DM2), based on atomic-level imaging studies of the defect, also conducted in their laboratory.