Boys with DMD continuously treated with eteplirsen for three years walked farther in six minutes than those receiving a placebo for the first six months
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...
MDA-supported Summit PLC announced it will continue testing it experimental drug in Duchenne MD patients, along with dietary modifications that may improve drug absorption
Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline
Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014.
PTC is moving forward with ataluren
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.
Neurologist Katherine Mathews discusses cardiac, respiratory and other concerns for people with limb-girdle muscular dystrophy
Neurologist Katherine Mathews
Katherine Mathews, M.D., is a neurologist at the University of Iowa Hospitals & Clinics in Iowa City, where she co-directs the MDA clinic. She serves on MDA's Medical Advisory Committee. Below she discusses limb-girdle muscular dystrophy with MDA Medical and Science Editor Margaret Wahl.
MDA partnered with a French muscle disease association to host a symposium on advancing gene therapy for neuromuscular diseases
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).
10 boys with Duchenne muscular dystrophy continue to show stabilization of walking ability; further trial results will be announced at an MDA conference in late April
Eteplirsen, an experimental exon-skipping therapy designed to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show sustained benefit on walking distance through week 74 of a phase 2b, 12-person study.