September 10, 2020

Sarepta Therapeutics Provides Program Update for SRP-9001 Micro-Dystrophin Gene Therapy

Sarepta Therapeutics announced that it has completed a meeting (Type C) with FDA’s Office of Tissues and Advanced Therapies (OTAT), an office that is part of the Center for Biologics Evaluation and Research (CBER). The…

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September 10, 2020

2020/2021 Flu Season: Stay Safe, Informed, and Healthy

As flu season approaches, we want to help you and your family stay safe, healthy, and informed. New “models” of flu appear each year. The Center for Disease Control and Prevention (CDC) examines the influenza strains that…

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September 7, 2020

It’s World Duchenne Awareness Day!

It’s World Duchenne Awareness Day! Today, September 7, is World Duchenne Awareness Day (#WDAD2020)! On this day we raise awareness for Duchenne around the globe. Each year, World Duchenne Awareness Day highlights a special theme that…

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September 3, 2020

10 Tips & Tricks for Navigating School & Home Routines

In response to the COVID-19 pandemic, many families in our community are facing changes in school and home routines. There can often be heightened emotions around these disruptions, leaving adults and kids alike stressed and…

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September 2, 2020

WATCH: NS Pharma Announcement of VILTEPSO (viltolarsen) – Webinar Recording

NS Pharma joined PPMD for a webinar on August 26, 2020 to provide an update about VILTEPSO™ from Ben Yungher, PhD, NS Pharma’s Medical Science Liaison, and its associated patient support program from Randy McGonigal,…

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September 2, 2020

Santhera Exercises Option to Obtain Worldwide Rights to Vamorolone in Duchenne and All Other Indications

We congratulate Santhera as they have exercised the option to obtain worldwide rights to vamorolone in Duchenne and all other indications. The license gives Santhera worldwide rights to vamorolone which means Santhera is responsible for…

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September 1, 2020

September is Duchenne Action Month!

SEPTEMBER IS DUCHENNE ACTION MONTH! This year’s Duchenne Action Month feels different, like everything, in light of the global pandemic. Under normal circumstances, everyone would be back at school or heading back to school –…

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August 26, 2020

PPMD’s 2020 Back-To-School Webinar Series – Resources & Takeaways

PPMD’s 2020 Back-To-School Webinar Series – Resources & Takeaways As we look towards this next school year, PPMD recognizes that our community is facing new and difficult choices about in-person and virtual learning. To help…

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August 25, 2020

Sarepta Announces FDA Acceptance of Casimersen NDA for Patients with Duchenne Amenable to Skipping Exon 45

PPMD is excited to learn that the FDA has accepted Sarepta’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045) and provided a regulatory action date of February 25, 2021. Casimersen is a potential…

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August 24, 2020

Learnings from PPMD’s COVID-19 Pandemic Survey

In spring 2020, Parent Project Muscular Dystrophy (PPMD) released a survey to better understand the patient experience with care, trials, and access to approved therapies during the COVID-19 pandemic. Thank you to everyone who contributed…

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