News Archive • World Duchenne

Showing all 30 results

The position of neuromuscular patients in Shared-Decision-Making

February 29, 2020

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

February 13, 2020

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

February 13, 2020

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)

February 5, 2020

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

January 29, 2020

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

December 24, 2019

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.

December 23, 2019

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 13, 2019

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

December 13, 2019

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

December 12, 2019

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

VISION-DMD Working Group Meeting

December 12, 2019

Athens, 6 and 8 December 2019 – The Vision-DMD Working Group Meeting organized by the World Duchenne Organization, preceded the…

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

December 11, 2019

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

November 13, 2019

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

November 12, 2019

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

Roche terminates SPITFIRE trial

November 6, 2019

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

October 14, 2019

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

August 6, 2019

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

April 19, 2019

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

April 10, 2019

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

Share4Rare launches community platform

April 2, 2019

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)

March 29, 2019

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

WDO Member Meeting 2019

March 14, 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

March 6, 2019

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

WDO Member Meeting 2019

February 24, 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…

Update on Duchenne UK’s survey and workshop to discuss the use of re-purposed or off label medicines

February 9, 2019

Last month, Duchenne UK hosted a workshop to discuss the use of re-purposed or off label medicines. We conducted a survey…

Translating DMD-Care Videos

February 6, 2019

Previous World Duchenne Awareness Day, we published a series of educational videos to make Duchenne Care information accessible for as…

PPMD Publishes 10 Year Registry Report

January 31, 2019

PPMD is excited to announce the release of the Ten Year Registry Report. This report is the culmination of data…

PPMD Duchenne Compass Meeting report

January 31, 2019

The Duchenne Patient-Focused Compass Meeting was held as part of FDA’s Patient-Focused Drug Development (PFDD) initiative. As an externally led…

PPMD Meeting Update: Inflammation in Duchenne

January 12, 2019

Last month, PPMD convened a day and half meeting – Inflammation in Duchenne: Current and New Treatment Strategies – to…

Project Hercules will receive Eurordis Company Award for Patient Engagement

November 14, 2018

In November 2017 Duchenne UK launched Project HERCULES to support access to new treatments for Duchenne Muscular Dystrophy (DMD). Today,…

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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

VISION-DMD Working Group Meeting

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

Update on Duchenne UK’s survey and workshop to discuss the use of re-purposed or off label medicines

Translating DMD-Care Videos

PPMD Publishes 10 Year Registry Report

PPMD Duchenne Compass Meeting report

PPMD Meeting Update: Inflammation in Duchenne

Project Hercules will receive Eurordis Company Award for Patient Engagement

DMD Emergency Program Provides Aid to Duchenne Families in Ukraine

Register for Duchenne Care Conference 2023

ADC open to accept applications for Duchenne Centers Accreditation