We are excited to announce that Habib Dystrophy Hope Foundation has joined the World Duchenne Organization as our newest Aspiring Member. Founded in 2020 by Mohammadi Mohiuddin, a devoted mother and advocate from Pakistan, the foundation is named to honor her beloved son, Habib, whose journey and resilience through Duchenne muscular dystrophy (DMD) continues to […]
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The Global DMD Adult Group, initiated by adults with Duchenne Muscular Dystrophy (DMD), aims to connect and empower people living with DMD and BMD. The goal is to become a platform for sharing stories, discussing challenges, while raising global awareness. Currently it is opening a call of interest to join the working group. Last World […]
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On September 14, the Becker Care Guidelines for the Clinical Management of Becker Muscular Dystrophy (BMD) were officially presented at the TREAT-NMD Becker Education and Engagement Day in Milan en Amsterdam in multiple languages. These guidelines are a major milestone. They represent the first clinical management tool specifically designed for patients and families affected by […]
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The documentary ‘Road to Independence’ portrays the lives of six young people living with neuromuscular diseases. Enrico is preparing to face his high school final exams and to say goodbye to the enriching experience of his life. Maddalena has left Sardinia, her homeland, to pursue her studies in Milan, embracing the challenges and opportunities of […]
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The recent BIND Webinar brought together patients, caregivers, and researchers with a shared interest in advancing our understanding of brain comorbidities in Duchenne and Becker muscular dystrophies (DMD/BMD). Hosted by the World Duchenne Organization, this webinar provided key insights into the BIND (Brain Involvement in Dystrophinopathies) Project, an international research initiative dedicated to studying learning […]
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World Duchenne Organization (WDO) and Duchenne Data Foundation (DDF) are thrilled to announce that applications for the Duchenne Patient Academy 2024 are now open for WDO Members. This online training event for Duchenne and Becker (DMD/BMD) patient advocates will take place online on December 6 and 7. This year we are inviting the new generation […]
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Prof. Annemieke Aartsma-Rus is taking on a challenge by reading and commenting on a paper a day. See below the overview of September 2024.
The post Monthly #apaperaday wrap-up: September 2024 appeared first on World Duchenne.
In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: The Adiponectin Receptor Agonist, ALY688: A Promising Therapeutic for Fibrosis in the Dystrophic Muscle Today’s pick is slightly older, from 2023 in @Dra_Cells by Dubuisson et al on testing an adiponectin receptor agonist in the mdx mouse model for Duchenne. Adiponectin is a […]
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In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Relationship between growth and ambulation loss in Duchenne muscular dystrophy boys on steroids Today I’m travelling to @Radboud_Uni to teach and the pick is from European Journal of Neurology by Stimpson et al on the relationship between growth and loss of ambulation in […]
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In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Evaluation of fatigue and fatigability in people with Duchenne muscular dystrophy using a dynamic arm support – a pilot study Today’s pick is again from Porto and the pilot test of fatigue and fatigability in Duchenne patients with and without using a dynamic […]
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Last month, families from around the world came together for PPMD Together: Virtual Edition. This unique virtual gathering provided families a space to connect, share their experiences, and find support from one another as they...
The post Catch up on PPMD Together: Virtual Edition – A Gathering of Families for Connection, Support, and Growth appeared first on Parent Project Muscular Dystrophy.
As I prepare for another holiday to begin, I am again overwhelmed with gratitude for the compassionate, resilient community we’ve built through PPMD. You are each an extension of my family, the family we have...
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Living with Duchenne and Becker presents unique challenges, especially for families from underserved or marginalized communities. Health disparities, delayed diagnoses, and limited access to care and clinical research create barriers that often result in diminished...
The post Equitable Access in Clinical Trials—A Call for Action and Collaboration appeared first on Parent Project Muscular Dystrophy.
REGENXBIO recently joined PPMD for a community webinar to provide an update on its AFFINITY DUCHENNE trial of RGX-202, REGENXBIO’s microdystrophin gene therapy. Dr. Naz Dastgir discussed the company’s pivotal trial plans and shared interim...
The post WATCH: RGX-202 – AFFINITY DUCHENNE Pivotal Program and Functional Data (Webinar Recording) appeared first on Parent Project Muscular Dystrophy.
PPMD commemorated its 30th anniversary with the “Investing in Every Future” celebratory event on November 14, 2024, in New York City. With some 200 attendees, this meaningful gathering honored three decades of progress and the resilience...
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REGENXBIO Inc. has announced that the AFFINITY DUCHENNE® open-label trial of RGX-202 has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety data from the Phase...
The post REGENXBIO Initiates Pivotal Phase of AFFINITY DUCHENNE® Trial of RGX-202 Gene Therapy, Reports Positive Functional Data appeared first on Parent Project Muscular Dystrophy.
Have you been looking for a way to engage with the community more? Do you have a personal experience that you would like to share with the larger community or through federal and state advocacy...
The post Calling All Adults with Duchenne & Becker: Join the 2025 PPMD Adult Advisory Committee appeared first on Parent Project Muscular Dystrophy.
Yesterday, REGENXBIO shared important updates on their Phase I/II AFFINITY DUCHENNE® trial of RGX-202 for Duchenne during their third quarter financial results update. RGX-202 utilizes a novel adeno-associated virus (AAV8) to transport a shortened version...
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Yesterday, Solid Biosciences shared new updates on the INSPIRE DUCHENNE clinical trial for their next-generation AAV micro-dystrophin gene therapy candidate, SGT-003, for the treatment of Duchenne. Based on early study results, the trial has expanded...
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Sarepta Therapeutics today announced that the company is discontinuing its SRP-5051 (vesleteplirsen) development program, including the global, Phase 2, multi-arm, ascending dose MOMENTUM study. Vesleteplirsen is an investigational, next-generation treatment utilizing Sarepta’s PPMO chemistry and...
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