Margarida Azevedo, MSc,  —

Capricor Therapeutics will hold a phone meeting with the U.S. Food and Drug Administration (FDA) this month to discuss potential product registration strategies for CAP-1002, a cardiac cell therapy being developed to treat Duchenne muscular dystrophy (DMD). CAP-1002 is produced from a healthy donor’s heart tissue, grown in a lab and stored…

An investigational therapy to treat congenital myotonic dystrophy, AMO-02 (tideglusib), has been granted fast track designation by the U.S. Food and Drug Administration (FDA), AMO Pharma has announced. Myotonic dystrophy (DM) is the most common form of muscular dystrophy (MD). DM type 1 is the consequence of a…

The U.S. Food and Drug Administration (FDA), which in September 2016 approved Serepta Therapeutics’ Duchenne muscular dystrophy (DMD) treatment Exondys 51 (eteplirsen), now finds itself the subject of a lawsuit aimed at forcing the FDA to turn over information about the approval process. Charles Seife, a journalism professor at New…

JAR of Hope, a foundation that funds Duchenne muscular dystrophy (DMD) research, is working to get legislation approved to make September 10-16 Duchenne Muscular Dystrophy Awareness Week in New York state. The foundation, along with New York City Assemblyman Michael Cusick, will ask New York Gov. Andrew M. Cuomo on…

Santhera Pharmaceuticals has updated the timeline for the ongoing assessment by the European Committee for Medicinal Products for Human Use (CHMP) of its Duchenne muscular dystrophy (DMD) drug, Raxone (idebenone). In June 2016, the European Medicines Agency’s (EMA) CHMP validated a marketing authorization application (MAA) for Raxone…

CureDuchenne has partnered with the Muscular Dystrophy Organization Nepal (MDON) to help deliver better care to Duchenne muscular dystrophy (DMD)  patients in that country, and education and support for their families. According to CureDuchenne, DMD affects over 300,000 children globally. An article, “Lived Experience of Mothers with Duchenne…

Summit Therapeutics has completed patient enrollment for PhaseOut DMD, a Phase 2 clinical trial evaluating the utrophin modulator ezutromid to treat Duchenne muscular dystrophy (DMD). PhaseOut DMD (NCT02858362) aims to provide proof-of-concept for ezutromid through measuring certain endpoints related to muscle structure, health and function. The trial could potentially provide…

The 7th Annual Getzlaf Golf Shootout to benefit CureDuchenne will take place Sept. 9 at Monarch Beach Golf Links in Dana Point, California. Brian Hayward will present the event, which will be hosted by the captain of the Anaheim Ducks hockey team, Ryan Getzlaf, and…

Japan has put Duchenne muscular dystrophy (DMD) drug candidate DS-5141b on its SAKIGAKE list, a designation to the U.S. Food and Drug Administration’s Fast Track Designation. United States’ fast tract. Daiichi Sankyo, the drug’s developer, announced the decision in a press release. An open-label Phase 1/2 clinical trial (NCT02667483), currently recruiting participants,…

The Duchenne muscular dystrophy (DMD) treatment Emflaza (deflazacort) was successfully acquired by PTC Therapeutics under terms agreed to in March, and may soon become available to patients at, potentially, a new and lower listing price. The now-completed agreement followed U.S. Food and Drug Administration (FDA) approval of Emflaza to treat all…