Development of treatments for Duchenne muscular dystrophy (DMD)continues to advance. Many of the new investigational drugs are potentially applicable to all DMD patients, while a few target those with specific mutations in the dystrophin gene.
Biopharmaceutical company Genervon has announced what it considers encouraging results from a "compassionate use" trial of its experimental drug GM604 in a single patient with advanced amyotrophic lateral sclerosis (ALS).
Boys with Duchenne muscular dystrophy (DMD) caused by specific genetic mutations who were treated with the experimental drug eteplirsen for 168 weeks (3.2 years) continued to show an advantage in the distance they were able to walk compared to those treated with the drug for only 144 weeks (2.8 years), although all boys had declined in walking distance since the start of this phase 2b trial of eteplirsen.
New Jersey-based PTC Therapeutics has begun submitting a rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of its experimental drug for the treatment of Duchenne muscular dystrophy (DMD) caused by specific flaws (mutations) in the dystrophin gene.
Results from a trial involving injection of genes for the follistatin protein into the thigh muscles of both legs in six men with Becker muscular dystrophy (BMD) show the experimental treatment appears safe and and was associated with improvement in walking ability (distance walked in six minutes) in four of the six trial participants.