News Archive - World Duchenne

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First reported case of COVID-19 in person affected by Duchenne

Last week, the news has reached us that a young Duchenne adult from Belgium has been infected with COVID-19. The…

Marketing authorization in Japan of VILTEPSO®

NS Pharma announced today that the Ministry of Health, Labour and Welfare (MHLW) has approved VILTEPSO® Intravenous Infusion 250 mg…

Raising awareness on psychosocial issues in DMD/BMD

Today, Duchenne Data Foundation launched a new website called Social Duchenne. This project aims to highlight psychosocial issues in people…

WDO Webinar 2 on COVID-19: on steroids, supplements and possible treatments

In weekly WDO Webinars, we ask DMD experts and clinicians to share their knowledge. Each time, we are collecting the…

FDA, EMA warns for fake medicines

Due to the new coronavirus outbreak, fraudulent business claim they have the cure to COVID are popping up rapidly. Both…

WDO Webinar 1: COVID-19 and DMD/BMD

This Saturday, the World Duchenne Organization hosted a webinar to facilitate the questions to clinicians on the current novel Coronavirus…

Live/ COVID-19: Coronavirus and Duchenne & Becker muscular dystrophy

A daily updated news feed on the COVID-19 outbreak with relevant information for Duchenne and Becker muscular dystrophy. March 30…

The position of neuromuscular patients in Shared-Decision-Making

In 2018, the European Neuromuscular Centre (ENMC) held a special meeting dedicated to the position of neuromuscular patients in Shared-Decisino-making…

La Force DMD joins the World Duchenne Organization

Since the official start of the World Duchenne Organization in 2007 (then named United Parent Projects Muscular Dystrophy), the number…

NDA accepted for filing by the FDA for antisense oligonucleotide Viltolarsen

NS Pharma announced the FDA has filed a New Drug Application (NDA) for their antisense oligonucleotide drug NS-065/NCNP-01 (viltolarsen). Viltolarsen is…

PTC Therapeutics publicises real-world data Translarna (ataluren)

PTC Therapeutics Announces First Publication of Real-World Data Showing TranslarnaTM (ataluren) Significantly Preserves Ability to Walk for Longer in Children…

Large international project to study dystrophin in the brain in Duchenne and Becker muscular dystrophy launched

The BIND project is the first project of this scale to improve characterisation of brain involvement in Duchenne and Becker Muscular Dystrophy (DMD…

Holiday wishes from WDO

2019 was a year where things have been given and taken from us. With this community letter, we hope to…

Roche provided rights to Sarepta’s investigational gene therapy for Duchenne.

Roche and Sarepta Therapeutics signed a licensing agreement providing Roche commercial rights to SRP-9001, Sarepta’s investigational gene therapy for Duchenne. …

FDA authorizes first test to aid in newborn screening for Duchenne Muscular Dystrophy

December 12, the U.S. Food and Drug Administration authorized marketing of the first test to aid in newborn screening for…

FDA approves VYONDYS 53TM (golodirsen) in the US

Sarepta Therapeutics Announces FDA Approval of VYONDYS 53TM (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients…

Duchenne Patient Academy trains 80 patient advocates from 37 countries

Three years ago, the first Duchenne Patient Academy (DPA) was launched in Athens, Greece. This global patient advocacy initiative was…

Prof. Annemieke Aartsma-Rus to receive EURORDIS Black Pearl Award 2020

11 December 2019, Paris – the World Duchenne Organization congratulates the recipients of the EURORDIS Black Pearl Awards 2020, announced today. Under…

WDO & PPMD certify the Czech Republic’s Motol Children’s Hospital

In late 2017, PPMD announced the formation of a Global Certified Duchenne Care Center (CDCC) Program, offering a path forward for…

Solid Biosciences IGNITE DMD placed on clinical hold

Official press release CAMBRIDGE, Mass., Nov. 12, 2019 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) today provided a clinical…

Roche terminates SPITFIRE trial

Unfortunately we have a very sad piece of news today: after an interim analysis of the SPITFIRE trial with Roche’s…

Open call for Duchenne Advocates

Context Over the past years, the Duchenne world has been changing due to a fast evolving research and regulatory landscape.…

Symposium On Muscle-Bone Interaction In DMD

21st June 2019 St Virgil Conference Centre, Salzburg, Austria On the 21st of June 2019 a symposium on muscle-bone interaction in…

WDO elected as European Patients' Forum member

The European Patients' Forum (EPF) is an umbrella organisation that works with patient groups in public health and health advocacy…

Highlights on Data sharing for Duchenne meeting

21-22 March 2019, Level Eleven – Amsterdam, the Netherlands Data is one of the few technologies that becomes of greater…

Share4Rare launches community platform

Share4Rare is born, an innovative platform to connect patients, caregivers and researchers of rare diseases around the globe. This multi-stakeholder project…

Duchenne Community Advisory Board (CAB)

Just over one year ago, Duchenne Data Foundation initiated the Duchenne Community Advisory Board (CAB) as part of the EuroCAB…

WDO Member Meeting 2019

The first WDO Member Meeting of 2019 was a great success. Over 50 participants from 24 countries gathered on February…

From Laboratory to Patient: an EMA Authorised Medicine Journey

The European Medicines Agency (EMA) is a neutral medicine regulator and can therefore not sponsor medicine, or fund research studies…

WDO Member Meeting 2019

During the annual Duchenne Conference organized by Parent Project Onlus that takes place in Rome on Feb 15-17, the World Duchenne Organization hosts a meeting…

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First reported case of COVID-19 in person affected by Duchenne

Marketing authorization in Japan of VILTEPSO®

Raising awareness on psychosocial issues in DMD/BMD

WDO Webinar 2 on COVID-19: on steroids, supplements and possible treatments

FDA, EMA warns for fake medicines

WDO Webinar 1: COVID-19 and DMD/BMD

Live/ COVID-19: Coronavirus and Duchenne & Becker muscular dystrophy

The position of neuromuscular patients in Shared-Decision-Making

La Force DMD joins the World Duchenne Organization

NDA accepted for filing by the FDA for antisense oligonucleotide Viltolarsen

PTC Therapeutics publicises real-world data Translarna (ataluren)

Large international project to study dystrophin in the brain in Duchenne and Becker muscular dystrophy launched

Holiday wishes from WDO

Roche provided rights to Sarepta’s investigational gene therapy for Duchenne.

FDA authorizes first test to aid in newborn screening for Duchenne Muscular Dystrophy

FDA approves VYONDYS 53TM (golodirsen) in the US

Duchenne Patient Academy trains 80 patient advocates from 37 countries

Prof. Annemieke Aartsma-Rus to receive EURORDIS Black Pearl Award 2020

WDO & PPMD certify the Czech Republic’s Motol Children’s Hospital

Solid Biosciences IGNITE DMD placed on clinical hold

Roche terminates SPITFIRE trial

Open call for Duchenne Advocates

Symposium On Muscle-Bone Interaction In DMD

WDO elected as European Patients' Forum member

Highlights on Data sharing for Duchenne meeting

Share4Rare launches community platform

Duchenne Community Advisory Board (CAB)

WDO Member Meeting 2019

From Laboratory to Patient: an EMA Authorised Medicine Journey

WDO Member Meeting 2019

PTC Therapeutics publicises real-world data Translarna (ataluren)

Large international project to study dystrophin in the brain in Duchenne and Becker muscular dystrophy launched

FDA approves VYONDYS 53TM (golodirsen) in the US