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July 23, 2018July 23, 2018

Raxone Delays Assisted Ventilation in DMD Patients by 3 Years, Phase 3 Trial Results Show

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Treatment of Duchenne muscular dystrophy (DMD) patients with Raxone (idebenone) delays the need for assisted ventilation by three years, new data from a Phase 3 clinical trial show. Trial ... Read more

July 20, 2018July 20, 2018

Axovant Enters Global Licensing Deal with Benitec for Investigational AXO-AAV-OPMD Program to Treat OPMD

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Benitec Biopharma recently granted Axovant Sciences worldwide rights to its investigational AXO-AAV-OPMD gene therapy program for the treatment of oculopharyngeal muscular dystrophy (OPMD) under a global licensing agreement. AXO-AAV-OPMD is ... Read more

July 18, 2018July 18, 2018

#PPMD2018 – Mexico Lags in Degree and Quality of Duchenne Care, Advocacy Groups Say

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Coping with Duchenne muscular dystrophy is difficult enough in the United States. But in neighboring Mexico, people with Duchenne often face social discrimination, lack of access to the newest ... Read more

July 18, 2018July 18, 2018

Acceleron’s ACE-083 Earns Orphan Drug Status for Facioscapulohumeral MD

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Acceleron’s ACE-083, for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA), the company announced. ... Read more

July 16, 2018July 16, 2018

#PPMD2018 — ‘Mitchell’s Journey’ Supports Duchenne Research, Helps Families Cope with Loss

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Five years after the death of a Utah boy named Mitchell Jones, the national foundation created in his name helps families struggling with the ravages of Duchenne muscular dystrophy. ... Read more

July 13, 2018July 13, 2018

Phase 3 Trial of Oral Edasalonexent as Potential DMD Treatment to Open Soon, Catabasis Announces

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A global Phase 3 trial evaluating the efficacy and safety of oral edasalonexent in patients with Duchenne muscular dystrophy (DMD) will begin enrolling boys, ages 4 to 7, in the coming ... Read more

July 12, 2018July 12, 2018

#PPMD2018 – Nebraska Mother of 3 Duchenne Boys: ‘We’ve Made a Choice to Be Positive’

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Betty Vertin had never heard of Duchenne muscular dystrophy before July 8, 2010 — the day a neurologist in Omaha, Nebraska, diagnosed her son Max with the rare disease. ... Read more

July 11, 2018July 11, 2018

#PPMD2018 – Don’t Overlook Dental and Digestive Issues, Duchenne Experts Advise

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With so much focus on the very real dangers of muscular, pulmonary and cardiac decline, it’s easy for moms and dads of Duchenne kids to forget about less urgent ... Read more

July 9, 2018July 10, 2018

Potential DMD Therapy Now in Phase 1 Trial, WVE-210201, Likely To Be Named Orphan Drug in EU

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The Committee for Orphan Medicinal Products (COMP), an arm of the European Medicines Agency (EMA), is recommending that WVE-210201 be designated an orphan drug as a potential Duchenne muscular dystrophy (DMD) ... Read more

July 9, 2018July 9, 2018

#PPMD2018 – Duchenne Research ‘Has Come a Long Way,’ PPMD Says at Meeting

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Never before has the therapeutic pipeline of potential treatments for Duchenne muscular dystrophy appeared so promising. Yet that also means more questions than ever. Searching for answers, about 620 people ... Read more

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