October 3, 2019

Updated Specifics of the 2019-2020 Influenza Vaccines

The flu virus is constantly changing, with new “models” of flu appearing each year. The Center for Disease Control and Prevention (CDC) looks at the strains of influenza that have affected Australia during their flu season, and…

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October 3, 2019

2019 World Muscle Society Update – Day 1

This week, PPMD’s Abby Bronson, SVP of Research Strategy and Eric Camino, PhD, Director of Research and Clinical Innovation are attending the annual World Muscle Society (WMS) meeting, this year in Copenhagen, Denmark. Below, Abby…

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October 2, 2019

NS Pharma Completes Rolling NDA Submission to FDA for NS-065/NCNP-01 (viltolarsen)

We are pleased to learn that NS Pharma has completed the submission of its rolling New Drug Application (NDA) to the FDA for NS-065/NCNP-01 (viltolarsen), which is under development for the treatment of Duchenne amenable…

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October 2, 2019

Can Girls Have Duchenne?

PPMD is often asked the question: Can girls have Duchenne? There are a number of Facebook groups focused on female carriers. Discussions and questions arise around why some female carriers of Duchenne have symptoms while…

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October 2, 2019

Duchenne UK and PPMD Award $200,000 Grant to Develop an Accepted Set of Patient Reported Outcomes

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK announced the joint funding of a project to create a set of agreed and validated Patient Reported Outcomes (PROs) for use in Europe and the US. The…

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October 1, 2019

First Baby Tested Via Parent Project Muscular Dystrophy’s Newborn Screening Pilot

Today, our community’s collective efforts have led to an incredible milestone for PPMD’s Duchenne Newborn Screening Pilot in New York State. Today, the first babies are being SCREENED for Duchenne. This is a significant milestone…

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October 1, 2019

Catabasis Completes Phase 3 PolarisDMD Trial Enrollment

Catabasis announced that the Phase 3 PolarisDMD trial for edasalonexent in Duchenne has completed enrollment and exceeded the target enrollment. PPMD is proud that The Duchenne Registry was able to play a role in helping…

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September 30, 2019

You can still join the fight to end Duchenne!

What an incredible Coach To Cure MD weekend! Our 12th anniversary season was our biggest to date with Werner Ladder joining the team as a national sponsor and Whataburger supporting us in the Southwest! With over 100…

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September 28, 2019

Today, we need YOU to Tackle Duchenne!

It’s GAMEDAY as we celebrate PPMD’s 12th Annual Coach To Cure MD, presented by Werner Ladder and we are so grateful to have teammates like you! Over 100 families and 450 universities are participating in the 12th…

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September 25, 2019

WATCH: Introduction to Gene Therapy [PPMD Gene Therapy Webinar Series]

For PPMD a pivotal aspect of care is education. Without the proper knowledge of what the potential benefits and risks are of an investigational product a family cannot make well informed decisions. As the landscape…

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