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.
In a May 22, 2014, letter, Prosensa provides updates on drisapersen and other Duchenne MD compounds in its pipeline, and on its natural history study
Dutch biopharmaceutical company Prosensa, developer of the experimental Duchenne muscular dystrophy (DMD) drug drisapersen, says it is moving the compound forward through regulatory agencies in the U.S. and Europe and will resume administering it to a first group of trial participants in the third quarter of 2014.
A phase 1b trial of a drug designed to increase utrophin protein levels in Duchenne MD-affected muscles, appears safe and may have muscle-protective effects
The European Medicines Agency expressed a 'positive opinion' on conditional approval for ataluren (Translarna) to treat Duchenne MD; a decision is expected within three months
The experimental Duchenne muscular dystrophy (DMD) drug ataluren (which has been given the brand name Translarna) has received encouragement from the European Medicines Agency (EMA) for conditional approval in European Union countries.
A pilot trial has found that one dose of tadalafil or sildenafil increased blood flow to Duchenne MD-affected muscles; a large tadalafil trial recently opened
A study conducted in 10 boys with Duchenne muscular dystrophy (DMD) has found that blood flow to exercising muscles is deficient and that treatment with either tadalafil (Cialis) or sildenafil (Viagra) normalizes this blood flow, at least in the short term (after one dose of either drug).
Tadalafil and sildenafil, both PDE5 inhibitors, are approved by the U.S. Food and Drug Administration (FDA)...
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.