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The recordings of Duchenne Care Conference 2024 are now available. It provides a platform for sharing the latest updates on Duchenne MD care.

The post Duchenne Care Conference 2024 successfully concluded appeared first on World Duchenne.

The World Duchenne Awareness Day 2024 theme is ‘Raise your voice for Duchenne’. World Duchenne Awareness Day (WDAD) is an annual event held on September 7. With this year’s theme, WDAD supports creating a society that provides equal opportunities for all. This year we organize the 11th edition.   Raise your voice for Duchenne This […]

The post World Duchenne Awareness Day 2024 Theme Announced appeared first on World Duchenne.

The World Duchenne Organization (WDO) is pleased to announce the release of a comprehensive White Paper titled ‘Newborn Screening for Duchenne Muscular Dystrophy: The Time is Now’ was published today.

The post White Paper Released: Newborn Screening for Duchenne Muscular Dystrophy – The Time is Now appeared first on World Duchenne.

From the start of May, Brazil is experiencing an environmental and humanitarian tragedy due to the floods. Nearly 1.5 million people are affected and many are Duchenne families who lost their home, some are isolated. The World Duchenne Organization has activated the DMD Emergency Program for the floods in Brazil. Karina Zuge, Chair of the […]

The post Tragic Flooding in Brazil: DMD Emergency Program activated appeared first on World Duchenne.

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Treatment with ataluren in four symptomatic Duchenne carriers. A pilot study #apaperaday brought to you again from #myologie2024 meeting in Paris. Today’s pick is from Acta Myology by Dori et al describing the experience with treating 4 female dystrophinopathy patients with ataluren (stop […]

The post #apaperaday: Treatment with ataluren in four symptomatic Duchenne carriers. A pilot study appeared first on World Duchenne.

It is with great sadness we share the information we just received. A patient participating in Pfizer’s Phase 2 DAYLIGHT study for Duchenne muscular dystrophy has passed away suddenly. Pfizer issued the following letter to the community. The patient received the investigational gene therapy, fordadistrogene movaparvovec, in early 2023.   Read the official statement by […]

The post Patient’s Sudden Death in Pfizer Gene Therapy Study appeared first on World Duchenne.

Prof. Annemieke Aartsma-Rus is taking on a challenge by reading and commenting on a paper a day. See below the overview of April 2024.

The post Monthly #apaperaday wrap-up: April 2024 appeared first on World Duchenne.

Registrations for the Duchenne Care Conference 2024 are now open. Healthcare professionals and patient organization representatives are invited to join this online educational event.

The post Duchenne Care Conference 2024: Registrations are open appeared first on World Duchenne.

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy #apaperaday from #Myologie2024 in Paris. Today’s pick is from @journal_nd by Zaidman et al on the management of adverse events after treatment with delandistrogene moxeparvovec (aka elevidys) […]

The post #apaperaday: Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy for Patients With Duchenne Muscular Dystrophy appeared first on World Duchenne.

In today’s #apaperaday, Prof. Aartsma-Rus reads and comments on the paper titled: Pharmacological inhibition of HDAC6 improves muscle phenotypes in dystrophin-deficient mice by downregulating TGF-β via Smad3 acetylation Today’s pick is another HDAC inhibition paper, which was featured already in 2022 as well. So this will be the first repeat tweet – we’ll do the […]

The post #apaperaday: Pharmacological inhibition of HDAC6 improves muscle phenotypes in dystrophin-deficient mice by downregulating TGF-β via Smad3 acetylation appeared first on World Duchenne.

Capricor Therapeutics has announced positive 3-year results from the ongoing HOPE-2 open-label extension (OLE) study of CAP-1002 for the treatment of Duchenne. CAP-1002 is the company’s novel cell therapy. The HOPE-2 is a double-blind, randomized,...

The post Capricor Therapeutics Announces Positive 3-Year Efficacy Results from HOPE-2 Open Label Extension Study of CAP-1002 appeared first on Parent Project Muscular Dystrophy.

NS Pharma, Inc. has shared preliminary analysis results from RACER53, the global Phase 3 clinical trial of NS-065/NCNP-01 (viltolarsen). Viltolarsen is an antisense oligonucleotide drug intended to treat patients with Duchenne who are amenable to...

The post NS Pharma Shares Update on VILTEPSO® (Viltolarsen) Phase 3 Study appeared first on Parent Project Muscular Dystrophy.

Dyne Therapeutics has shared new clinical data from the ongoing DELIVER trial of DYNE-251 in patients with Duchenne who are amenable to exon 51 skipping. DYNE-251 is an exon skipping product that combines a PMO...

The post Dyne Therapeutics Shares New Clinical Data from DELIVER Trial of DYNE-251 in Duchenne Patients appeared first on Parent Project Muscular Dystrophy.

PPMD is pleased to share that the article, “Barriers to diverse clinical trial participation in Duchenne muscular dystrophy: Engaging Hispanic/Latina caregivers and health professionals,” has been published in the Orphanet Journal of Rare Diseases. This...

The post Study to Explore Barriers to Diverse Clinical Trial Participation in Duchenne Published appeared first on Parent Project Muscular Dystrophy.

PTC Therapeutics, Inc. announced today that the European Commission (EC) has decided not to adopt the Committee for Medicinal Products for Human Use’s (CHMP) January 2024 negative opinion on the annual renewal of the conditional...

The post PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation appeared first on Parent Project Muscular Dystrophy.

This week marked a significant milestone as Congress successfully passed the bipartisan Federal Aviation Administration (FAA) reauthorization, officially known as HR 3935, the Securing Growth and Robust Leadership in American Aviation Act. This legislation, now...

The post Congress Passes FAA Reauthorization Featuring Monumental Accessibility Improvements appeared first on Parent Project Muscular Dystrophy.

Last week, PPMD held the 2024 Cardiac Care Workshop in Baltimore, Maryland, which included 40 academics and professionals and representatives from 11 industry partners to discuss how to move beyond the current state of cardiac...

The post Mapping Out a Path Forward: PPMD Convenes Representatives from 21 Academic Centers and 11 Industry Partners for 2024 Cardiac Care Workshop appeared first on Parent Project Muscular Dystrophy.

PPMD is proud to share some exciting news: our very own President and CEO, Pat Furlong, has been named the 2024 recipient of the prestigious Sonia Skarlatos Public Service Award by the American Society of...

The post PPMD’s Pat Furlong Honored with ASGCT’s Sonia Skarlatos Public Service Award appeared first on Parent Project Muscular Dystrophy.

Members of the PPMD community provided testimony today during the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) quarterly meeting, voicing support for adding Duchenne to the Recommended Uniform Screening Panel (RUSP) for...

The post Updates from the ACHDNC Meeting and Next Steps for Duchenne Newborn Screening appeared first on Parent Project Muscular Dystrophy.

PPMD is excited to bring together experts in the cardiac field, spanning clinicians and researchers, to the third annual PPMD Cardiac Workshop, May 9-10, 2024 to delve into current and emerging issues in cardiac care...

The post PPMD Hosts 2024 Cardiac Workshop, May 9-10 appeared first on Parent Project Muscular Dystrophy.