Quest Magazine

DMD: Eteplirsen Data Still Encouraging at 144 Weeks

Walking ability and respiratory function in boys with Duchenne muscular dystrophy (DMD) show continued benefit from eteplirsen at 144 weeks (almost three years), the drug's developer announced today. In addition, the intravenously infused drug was well tolerated, with no serious treatment-related adverse events seen.

Sarepta Therapeutics Reports Long-Term Outcomes from Phase IIb Study of Eteplirsen

MDA Invites Public To Vote For Their Favorite Telethon Moment

DMD: FDA Gives Fast Track Designation to HT-100

HT-100, an experimental drug being developed for Duchenne muscular dystrophy (DMD) with support from MDA, has shown preliminary safety and signs of the desired effects on scar tissue formation in the first 17 trial participants, and it has received "fast track" designation from the U.S. Food and Drug Administration (FDA).

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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