October 16, 2018

Behavior Challenges and Duchenne: Part 2 – Establishing Consequences

Psychosocial health (also called “mental health”) includes behavior, emotional adjustment, learning/school success, and relationships. These things are very important for overall well-being and quality of life. Addressing the psychosocial needs of a person with Duchenne is…

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October 15, 2018

WATCH: Is Myostatin Still a Potential Therapeutic Target? – Webinar Recording

Myostatin is a negative regulator of muscle growth – a protein that stops muscle building and doesn’t let them get too big, as part of the natural regeneration process. In other words, if we can…

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October 11, 2018

Pediatrics Supplement Focuses on Specialty Care for People with Duchenne

The supplement “Specialty Care for the Patient with Duchenne Muscular Dystrophy” was recently published in the October 2018 issue of Pediatrics. The supplement aims to raise the standard of care and improve quality of life…

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October 9, 2018

2018 World Muscle Society Update

Last week, PPMD’s Founding President and CEO, Pat Furlong and our SVP of Research Strategy, Abby Bronson attended the annual World Muscle Society (WMS) meeting, this year in Mendoza, Argentina. Pat and Abby have provided…

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October 5, 2018

NS Pharma Announces Positive Results of US/Canada Phase II Clinical Trial of Viltolarsen at World Muscle Society

NS Pharma announced positive results of a US/Canada Phase II clinical trial of viltolarsen at the World Muscle Society meeting held in Mendoza, Argentina. The preliminary findings suggest clinical improvement as measured by timed function…

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October 4, 2018

Jerry Mendell, M.D. Shares Positive Updated Results from Gene Therapy Micro-dystrophin Trial

  PPMD is excited to learn that Dr. Jerry Mendell of Nationwide Children’s Hospital today shared positive results for the fourth patient in Sarepta’s micro-dystrophin trial, indicating positive functional signals for all of the patients….

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October 3, 2018

WATCH: Puberty and Duchenne – Webinar Recording with Dr. Robert Benjamin, Pediatric Endocrinologist

Adolescence is a time of change – changing bodies, changing emotions, changing friends, changing lives. All of these changes are part of puberty, caused by increased levels of testosterone (the male sex hormone). But for…

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October 2, 2018

Advocacy Matters! Duchenne Community Requests Included in 2019 Federal Budget

Last week, the President signed the FY19 Labor Health and Human Services funding bill which included nearly all provisions advocated by this community early in the year. Your calls, your emails, your face to face…

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October 1, 2018

New Duchenne/Becker ICD Code in Effect Today — G71.01

Today at 12:01 AM  – while most of us were sound asleep – the International Classification of Diseases (ICD) code for Duchenne/Becker muscular dystrophy changed.  Previously, Duchenne muscular dystrophy was classified among a broad category…

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September 30, 2018

You can still join the fight to end Duchenne!

As we reflect on yesterday’s Coach To Cure MD success at colleges and high schools across the country, I am once again taking a moment to revel in what we do as a community. Last year, PPMD…

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