News Archive - World Duchenne

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Santhera completes enrollment of Phase 3 SIDEROS Study with Puldysa® (Idebenone)

Today, Santhera Pharmaceuticals announced full recruitment of its Phase 3 SIDEROS study with idebenone in Duchenne muscular dystrophy (DMD). The…

Updated clinical data on Pfizer's phase 1b gene therapy study

Pfizer today announced updated clinical data on PF-06939926, an investigational gene therapy being developed to treat Duchenne muscular dystrophy (DMD).…

Capricor announces HOPE-2 Study results

The 12-month data from HOPE-2 showed statistically meaningful improvements in the PUL 2.0 in CAP-1002 treated patients (p=0.05) with a…

Catabasis: update on edasalonexent

Catabasis shared today their Catabasis Connection newsletter, with updates on edasalonexent and information shared at the MDA Virtual Poster Session.…

IRDiRC publishes Orphan Drug Development Guidebook

Over the past 2-3 years, IRDiRC has been working with their Therapies Scientific Committee, on a project called Orphan Drug Development…

UPDATE ON CLINICAL HOLD IGNITE DMD

This morning, Solid Biosciences issued a press release following receipt of written communication from the FDA regarding the clinical hold placed…

Caring for people with Duchenne, Becker and other MD's during COVID-19

COVID-19 has shaken up the healthcare world. Delivering clinical care to people affected with Duchenne, Becker and other muscular dystrophies…

Italfarmaco update on Givinostat

Update on advanced-stage clinical development program with Givinostat. The Italfarmaco Group provided today an update on the clinical development program…

How to optimise your social media channels to receive donations

Over the last few weeks we have seen many events like conferences and fundraising activities are being postponed or even…

ECRD 2020 Online Conference – registration opened

EURORDIS, together with Orphanet and Inserm are hosting their 10th European Conference on Rare Diseases & Orphan Products (ECRD) on…

Webinar 4: how to manage COVID-19 induced stress and anxiety

Even under the best circumstances, it’s hard to take care of a child with a chronic disease. It’s even harder…

WDO Webinar 3: how to prepare for potential DMD/BMD patients with COVID-19

What is the best we can do for our Duchenne and Becker patients? We all wish to provide the best…

Autism in Duchenne muscular dystrophy: how to detect and what to do

Thursday April 2 is World Autism Awareness Day. Then, the world raises awareness about people with Autism Spectrum Disorder (ASD)…

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 regularly updated news feed on the COVID-19 outbreak with relevant information for Duchenne and Becker muscular dystrophy. May 9…

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…

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Santhera completes enrollment of Phase 3 SIDEROS Study with Puldysa® (Idebenone)

Updated clinical data on Pfizer's phase 1b gene therapy study

Capricor announces HOPE-2 Study results

Catabasis: update on edasalonexent

IRDiRC publishes Orphan Drug Development Guidebook

UPDATE ON CLINICAL HOLD IGNITE DMD

Caring for people with Duchenne, Becker and other MD's during COVID-19

Italfarmaco update on Givinostat

How to optimise your social media channels to receive donations

ECRD 2020 Online Conference – registration opened

Webinar 4: how to manage COVID-19 induced stress and anxiety

WDO Webinar 3: how to prepare for potential DMD/BMD patients with COVID-19

Autism in Duchenne muscular dystrophy: how to detect and what to do

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

Caring for people with Duchenne, Becker and other MD's during COVID-19

Italfarmaco update on Givinostat

Autism in Duchenne muscular dystrophy: how to detect and what to do