Summit Therapeutics plc announced today that it has enrolled its first patients at trial sites in the US into PhaseOut DMD, a Phase 2 clinical trial of ezutromid in Duchenne. The 48-week, open-label (meaning all participants receive ezutromid) Phase 2 trial is enrolling up to 40 boys total between their 5th…

Frequently, I get calls from mothers/sisters/aunts of people living with Duchenne about issues related to being a carrier of Duchenne:

• Should they have carrier testing?
• Why should they have carrier testing? 
• If they are…

PTC Therapeutics, Inc. today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna(TM) (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients…

Everyone, six months of age and older, needs to be vaccinated against influenza (flu), and protecting people living with with Duchenne is especially important. A 2005 study done by the…

In February, dosing and new patient enrollment in all cohorts of the HALO trial, a study evaluating the compound HT-100 in patients with Duchenne, was suspended after one of the patients in the trial, receiving 60μg/kg/d (the highest dose in the study), passed away after experiencing serious, life-threatening health issues. Since then, the company has been working with the…

Pfizer has provided the following update regarding the on-going Phase II clinical trial of domagrozumab (PF-06252616), a myostatin inhibitor, in people with Duchenne.

Notably, the age range for this clinical study has been expanded from 6 to <10 years of age to 6 to <16 years of age. Additional sites have been added in the US, United Kingdom, Poland, Bulgaria, and Australia.



Pfizer also reports that enrollment of the phase 2 study of…

With the accelerated approval of EXONDYS 51™ (aka eteplirsen) by the FDA in September, the Duchenne community has been quickly introduced to a whole new vocabulary and a whole new list of concerns. We knew…