Drugs

A look at symptoms, diagnosis, treatments and current MDA-supported research into this complex autoimmune disorder

posted on June 15, 2015 - 1:01pm
In recognition of June being National Myasthenia Gravis Awareness Month, MDA is examining the disease from several perspectives. This article offers an overview of signs, symptoms, diagnosis and treatments for myasthenia gravis (MG), as well as information on drugs that people with MG should avoid.

Neuromuscular disease specialist and biotechnology company employee Timothy Miller says it can be a significant risk and financial hardship for a small company to supply a drug that has not been through the full approval process

posted on July 7, 2014 - 9:09am
Quest Summer 2014
Timothy Miller has been a university-associated neurologist specializing in neuromuscular disorders who has recently moved to the biotechnology industry. He has been the director of the MDA-supported pediatric neuromuscular disorders clinic at Children's Clinics for Rehabilitative Services in Tucson, Ariz.; an assistant professor of neurology, pathology and pediatrics at the University of Arizona...

A trial of an experimental compound to counteract muscle inflammation and scarring in Duchenne MD resumes, after an interruption to complete a dog safety study

posted on June 17, 2014 - 12:38pm
Update (July 3, 2014): Akashi says the HT-100 main trial is now closed; the extension study is open to those who have already participated in the main trial.

Isis Pharmaceuticals is launching a phase 1 trial of ISIS-DMPKRx, an experimental drug for type 1 myotonic muscular dystrophy

posted on June 10, 2014 - 8:30am
  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...

The company plans to submit a New Drug Application to the U.S. Food and Drug Administration later this year, and has committed to conducting two confirmatory post-approval clinical trials

posted on June 6, 2014 - 9:56am
Dutch biopharmaceutical company Prosensa has outlined plans to seek accelerated approval in the United States for its experimental drug drisapersen, under development for the treatment of Duchenne muscular dystrophy (DMD). The company will seek approval for the drug in Europe as well.

Santhera Pharmaceuticals' investigational drug to treat respiratory dysfunction in Duchenne muscular dystrophy has shown benefit compared to a placebo in a phase 3 trial

posted on May 29, 2014 - 10:23am
Results from a phase 3, 65-participant trial of idebenone (brand names are Catena and Raxone) in boys with Duchenne muscular dystrophy (DMD) show the drug reduced the decline in respiratory function compared to a placebo, potentially paving the way toward regulatory approval.

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

posted on May 28, 2014 - 8:12am
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.

Sarepta Therapeutics says the U.S. Food and Drug Administration has changed its view of a possible accelerated approval pathway for Duchenne MD drug eteplirsen

posted on November 12, 2013 - 2:46pm
Update (Dec. 10, 2013: Sarepta has posted webcasts of a presentation given Dec. 2, 2013, at a Deutsche Bank BioFEST conference; and of a presentation given Dec.