Quest Magazine

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.

Sailormen Inc. Popeyes Raises Record $703,034 For Local MDA Families

UPDATE: DMD: Encouraging Results for SMT C1100

 

DMD: Ataluren May Get Conditional Approval in Europe

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.

MDA Ride for Life XXVII Raises More Than $1 Million for the Fight Against Muscle Disease

Sarepta Therapeutics Announces Agreement for Acquisition of Manufacturing Facility in Massachusetts

DMD: Sarepta Will Pursue Accelerated Approval for Eteplirsen and Conduct Additional Trials

Update (May 27, 2014): Sarepta has acquired a manufacturing facility in Massachusetts to enhance its ability to produce investigational exon-skipping therapies DMD. In a May 22 press release, Sarepta CEO Chris Garabedian said, "While we scale up to address the potential U.S.

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