Quest Magazine

DMD: HT-100 Trial Has Reopened

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.

MDA Deeply Saddened by Passing of Broadcasting Legend Casey Kasem

Improving Drug Development: The MDA Community Weighs In

Last month, the Energy & Commerce Committee of the U.S. House of Representatives launched a bipartisan effort called the 21st Century Cures initiative to accelerate the process by which drugs are discovered and delivered to patients. Congress has sought input from the patient community and advocacy organizations like MDA.

ALS: BrainStorm to Test NurOwn Cells in US

MDA Moves into New Chicago National Office

MMD1: Antisense Drug Moves to Human Testing

 

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.

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Isis Pharmaceuticals Initiates Phase 1 Study of ISIS-DMPK Rx to Treat Myotonic Dystrophy Type 1

Isis Earns $14 Million Milestone Payment from Biogen Idec ISIS-DMPKRx is the Second Generation 2.5 Antisense Drug to Enter the Clinic
 

Prosensa Will Pursue Accelerated Approval for Drisapersen to Treat DMD

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.

DMD: Idebenone Slows Decline of Respiratory Function

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.

DMD: Drisapersen Dosing Planned for Later in 2014

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.

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