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November 2016 Blog Posts (8)


Staff
A truly thankful moment

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Added by Pat Furlong on November 23, 2016 at 5:30pm — No Comments


Staff
Summit Enrolls U.S. Patients into Phaseout DMD, a Phase 2 Clinical Trial of Ezutromid in Duchenne

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…

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Added by PPMD on November 16, 2016 at 9:00am — No Comments


Staff
Duchenne Carrier Study at Nationwide Children’s Hospital

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…
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Added by Kathi Kinnett on November 14, 2016 at 11:00am — 2 Comments


Staff
CHMP Recommends Renewal of Translarna's™ Marketing Authorization Based on Continued Positive Benefit-Risk Assessment

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…

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Added by PPMD on November 11, 2016 at 9:30am — No Comments


Staff
2016 Flu Vaccination Recommendations

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 …

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Added by Kathi Kinnett on November 9, 2016 at 2:30pm — 2 Comments


Staff
Akashi Provides HT-100 Clinical Trial Update

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…

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Added by PPMD on November 7, 2016 at 11:30am — No Comments


Staff
Pfizer Shares Update on Phase II Clinical Trial of Domagrozumab (PF-06252616) in Duchenne

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…

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Added by PPMD on November 2, 2016 at 2:30pm — No Comments


Staff
Understanding Insurance Determinations



With the accelerated approval of EXONDYS 51™ (aka eteplirsen) by the FDA in September, the Duchenne community has been quickly…

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Added by Annie Kennedy on November 2, 2016 at 12:30pm — No Comments

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