December 1, 2023

Watch: AGAMREE® Approval with Catalyst Pharmaceuticals, Inc. (Webinar Recording)

PPMD was recently joined by Catalyst Pharmaceuticals to review the recent FDA approval of AGAMREE (vamorolone), a new steroid approved for the treatment of Duchenne.  Amy Grover, Catalyst’s Senior Director of Patient Advocacy, introduced the…

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December 1, 2023

Empowering Carriers of Duchenne: New Carrier Publications Resource

Being a carrier of Duchenne can feel isolating and misunderstood, especially for those who manifest symptoms. Earlier this year, PPMD began holding monthly support meetings for manifesting carriers to talk through their experiences, raise awareness,…

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November 29, 2023

United Nations Officially Designates September 7 as World Duchenne Awareness Day

The United Nations has marked a momentous milestone for the global rare disease community by officially designating September 7th as World Duchenne Awareness Day, set to be observed annually from 2024. The adoption of the…

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November 29, 2023

Italfarmaco Announces PDUFA Extension for the FDA’s Review of Givinostat

Italfarmaco Group announced an extension in the FDA’s review of the New Drug Application (NDA) for Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor for the potential treatment of Duchenne. The new Prescription Drug User…

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November 28, 2023

Every donation made for #GivingTuesday will be DOUBLED

On this #GivingTuesday, let’s unite in the fight for the future of those living with Duchenne. Your donation today holds extraordinary power as we have a unique opportunity to DOUBLE its impact! The Alex Rezkalla…

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November 23, 2023

This Year, I Am Grateful for the Monumental Impact We’ve Made Together

Thanksgiving offers each of us a moment to reflect on all that we are grateful for. This year, I am filled with gratitude for the impact we have made on the Duchenne landscape these past…

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November 20, 2023

CDCC Spotlight: Children’s Hospital of Richmond at VCU

Focusing on the Needs of Young Adults Medical appointments for adults living with Duchenne & Becker can look very different than the care they receive as pediatric patients. At the Certified Duchenne Care Center at…

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November 16, 2023

Fight for Every Future by Supporting PPMD’s Gene Therapy Initiative

In 1984, the word “Duchenne” entered my world, a life-changing moment when doctors could only offer the advice to go home and love my sons because they believed nothing more could be done. Since that…

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November 16, 2023

PPMD Urges UN Support for ‘World Duchenne Awareness Day’ Resolution Ahead of UN General Assembly Meeting

PPMD CEO and President Pat Furlong and Buddy Cassidy spoke yesterday in front of 60+ diplomats to encourage ambassadors to the United Nations and members of the World Health Organization to support a draft resolution…

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November 14, 2023

Solid Biosciences Receives FDA Clearance for Investigational New Drug Application for SGT-003 Gene Therapy Candidate

PPMD is pleased to learn that Solid Biosciences has received clearance by the U.S. Food and Drug Administration (FDA) for an Investigational New Drug (IND) application for SGT-003, the company’s next generation microdystrophin gene therapy…

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