February 16, 2021

Save the Date for PPMD’s Annual Conference: Virtual Edition – June 23-26, 2021

PPMD’s Annual Conference – Virtual Edition June 23-26, 2021 It’s hard to believe that we are still in this pandemic. We are getting closer to safer days ahead and we want our Duchenne and Becker…

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February 12, 2021

Meet the 2021 PPMD Adult Advisory Committee (PAAC)

PPMD is thrilled to announce the members of the 2021 PPMD Adult Advisory Committee (PAAC)! PAAC members serve at a leadership capacity to represent the teen and adult Duchenne and Becker community. Despite the inability…

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February 11, 2021

A DIY Virtual Walk Success Story

Rally4RJ Virtual Walk in the Park: A DIY Virtual Walk Success Story! Suzanne Sullivan and her family were excited to hold their 2nd annual Rally4RJ 1 Mile Walk in the Park in the summer of…

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February 10, 2021

“See you on the Road”: PPMD’s End Duchenne Tour 2021 Stops

Stemming from our PPMD’s Connect Roundtables, PPMD’s End Duchenne Tour stops were formally established in 2015 as the Every Single One Tour. Tour stops are typically one-day long events, providing updates on the latest clinical…

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February 10, 2021

Gene Therapy and Immune Response: Understanding Challenges and Strategies

PPMD recently announced the formation of the Pediatric Gene Therapy and Medical Ethics (PGTME) Working Group to address the challenges involved in gene therapy. As part of their recent educational series, the PGTME held a…

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February 9, 2021

PPMD Receives $100,000 Award from Duchenne Research Fund to Address Neurobehavioral Issues in Duchenne

Parent Project Muscular Dystrophy (PPMD) is excited to announce sponsorship from the Duchenne Research Fund for an award totaling over $100,000. As part of PPMD’s new Brain Initiative, the award will support work in understanding…

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February 5, 2021

PTC Shares Results of Study 045 of Translarna™ (Ataluren) in Patients with Nonsense Mutation Duchenne

Twenty years ago, we were first introduced to PTC Therapeutics. We watched as PTC-124 was in early studies of safety and dose finding. Over the years, as studies continued, PTC-124 was referred to as ataluren…

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February 4, 2021

WATCH: Planning For Your Future – Navigating Benefits & Finances

Last week we were joined by Amber Sapp, parent, and Leona Filis, attorney, to discuss planning for your family’s future in regards to benefits and finances. Although these subjects can feel overwhelming, Amber and Leona…

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February 3, 2021

TODAY: Virtual Town Hall at 1 PM EST

Advocacy in Action: Virtual Town Hall Facebook Live | February 3, 2021 To commemorate the passage of the MD-CARE Act, PPMD will host a Virtual Town Hall on Wednesday, February 3, 1 PM – 4…

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February 1, 2021

Santhera Shares Community Update on Phase 2b Trial for Vamorolone

Santhera Pharmaceuticals has shared an update with the community regarding the phase 2b trial for vamorolone, which the company expects to have data from in the second quarter of 2021. The update includes contact information…

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