Israel-based BioBlast Pharma says a California site will be added soon to locations already testing Cabaletta in oculopharyngeal muscular dystrophy in Israel and Canada
BioBlast Pharma, based in Tel Aviv, Israel, has been given clearance from the U.S. Food and Drug Administration (FDA) to test its experimental drug Cabaletta in the U.S. in people with oculopharyngeal muscular dystrophy (OPMD).
Stanford University is collecting samples of blood, muscle and other tissues from people with neuromuscular disorders for use in research
Ever wondered how someone with a neuromuscular disorder in his or her family might contribute to research efforts in this field?
Research updates and clinical trials information
What About My Disease?
Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.
Israeli biotechnology company BioBlast is developing and testing Cabaletta, designed to counteract abnormal clumping of cellular proteins in oculopharnyngeal muscular dystrophy (OPMD)
Update (March 17, 2015): In a March 12, 2015, press release, BioBlast announced it will open a site for the Cabaletta in OPMD study in California during the second quarter of 2015.
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.