News Archive - World Duchenne

Showing all 30 results

Attention to mental well-being in DMD/BMD families
10/10/2020

Living with Duchenne and Becker muscular dystrophy (DMD and BMD) has a profound impact on the lives and mental health…

Santhera stops Phase 3 SIDEROS Study and Development of Puldysa®
06/10/2020

Today, Santhera announced the discontinuation of its Phase 3 SIDEROS study with Puldysa® (idebenone) in patients with Duchenne muscular dystrophy…

New Russian patient organization 'CF Gordey' joins WDO
22/09/2020

CF Gordey is a family created patient advocacy organization who is working with families, healthcare, society and the state to…

Duchenne and the brain: What is known from animal models?
07/09/2020

We all know that in Duchenne and Becker muscular dystrophy, the muscles are affected. However, there are also dystrophin variants…

Duchenne and the Brain
07/09/2020

An online program about brain involvement in Duchenne and Becker muscular dystrophy will launch on World Duchenne Awareness Day. This…

Santhera tries to get worldwide rights to Vamorolone for all indications – including DMD
02/09/2020

Santhera exercises the option to obtain worldwide rights of Vamorolone in Duchenne muscular dystrophy and all other indications. Santhera will…

FDA accepts review of casimersen for DMD patients amenable to skipping exon 45
25/08/2020

Today, Sarepta announced the FDA accepted Casimersen for DMD patients amenable to skipping exon 45. The US Food and Drug Administration…

Exploring preclinical and clinical studies that might not have met expectation
20/08/2020

The World Duchenne Organization is happy to hear that Duchenne Parent Project NL and Parent Project MD are supporting the…

FDA grants accelerated approval for Viltepso (viltolarsen)
12/08/2020

Nippon Shinyaku unit NS Pharma today announced the accelerated FDA for Viltepso (viltolarsen). This targeted treatment is created for people…

EJP RD – Networking Support Scheme (NSS) Call
12/08/2020

The goal of the Networking Support Scheme (NSS) in the European Joint Programme for Rare Diseases (EJP RD) is to…

Webinar: Returning clinical research results to trial participants
11/08/2020

VISION-DMD Webinar: Wednesday 19th August 2020 3pm - 4pm CEST Children and their families participating in clinical trials have invested…

Fast Track Designation for Sarepta's Gene Therapy
26/07/2020

Sarepta announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). SRP-9001 is…

Patient centered and interoperable registry hub for Rare Neuromuscular Diseases
14/07/2020

EURO-NMD, the European Reference Network for Rare Neuromuscular Diseases, spans 14 European countries, with 61 reference centres that oversee more…

#WDAD2020: Together we are stronger
14/07/2020

The current Corona pandemic is profoundly impacting our daily lives. It changes the way we spend time, and it alters…

CHMP provides Translarna™ (ataluren) Label Update
01/07/2020

Translarna is currently indicated for ambulatory Duchenne patients who are over two years of age; the requested extension would have…

Sarepta completes NDA submission for Casimersen
26/06/2020

Sarepta today announced they have completed the submission of a rolling New Drug Application (NDA) to the U.S. Food and…

Care in times of COVID
26/06/2020

Over the past months, we have been receiving an increasing number of questions about the risks of returning, or not…

WDAD 2020: Duchenne and the brain
17/06/2020

Every year on September 7, we celebrate World Duchenne Awareness Day. On this day, we raise awareness for Duchenne and…

Sarepta announces positive safety and efficacy data from gene therapy trial
15/06/2020

Sarepta shared a press release this morning. In here, Sarepta Therapeutics announces positive safety and efficacy data from the SRP-9001…

EC grants Orphan Drug Designation to viltolarsen
09/06/2020

Today, Nippon Shinyaku announced that the European Commission (EC) has granted Orphan Drug Designation to viltolarsen. Viltolarsen is an antisense…

Regulatory Review of Santhera's Idebenone by the CHMP
29/05/2020

Santhera announced today that the EMA’s CHMP has granted a requested extension of the clock-stop in the regulatory procedure for…

Finland establishes first Duchenne patient organization
29/05/2020

On Benjamin's first birthday, the Finell family received a phone call from the hospital that not only Benjamin but also…

NS Pharma Announces Publication of Clinical Trial Data for Viltolarsen
27/05/2020

NS Pharma, a wholly owned subsidiary of Nippon Shinyaku, announced that JAMA Neurology has published results from a clinical trial…

Santhera completes enrollment of Phase 3 SIDEROS Study with Puldysa® (Idebenone)
20/05/2020

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
15/05/2020

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
13/05/2020

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
13/05/2020

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
08/05/2020

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
07/05/2020

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
01/05/2020

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

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Attention to mental well-being in DMD/BMD families
10/10/2020

Santhera stops Phase 3 SIDEROS Study and Development of Puldysa®
06/10/2020

New Russian patient organization 'CF Gordey' joins WDO
22/09/2020

Duchenne and the brain: What is known from animal models?
07/09/2020

Duchenne and the Brain
07/09/2020

Santhera tries to get worldwide rights to Vamorolone for all indications – including DMD
02/09/2020

FDA accepts review of casimersen for DMD patients amenable to skipping exon 45
25/08/2020

Exploring preclinical and clinical studies that might not have met expectation
20/08/2020

FDA grants accelerated approval for Viltepso (viltolarsen)
12/08/2020

EJP RD – Networking Support Scheme (NSS) Call
12/08/2020

Webinar: Returning clinical research results to trial participants
11/08/2020

Fast Track Designation for Sarepta's Gene Therapy
26/07/2020

Patient centered and interoperable registry hub for Rare Neuromuscular Diseases
14/07/2020

#WDAD2020: Together we are stronger
14/07/2020

CHMP provides Translarna™ (ataluren) Label Update
01/07/2020

Sarepta completes NDA submission for Casimersen
26/06/2020

Care in times of COVID
26/06/2020

WDAD 2020: Duchenne and the brain
17/06/2020

Sarepta announces positive safety and efficacy data from gene therapy trial
15/06/2020

EC grants Orphan Drug Designation to viltolarsen
09/06/2020

Regulatory Review of Santhera's Idebenone by the CHMP
29/05/2020

Finland establishes first Duchenne patient organization
29/05/2020

NS Pharma Announces Publication of Clinical Trial Data for Viltolarsen
27/05/2020

Santhera completes enrollment of Phase 3 SIDEROS Study with Puldysa® (Idebenone)
20/05/2020

Updated clinical data on Pfizer's phase 1b gene therapy study
15/05/2020

Capricor announces HOPE-2 Study results
13/05/2020

Catabasis: update on edasalonexent
13/05/2020

IRDiRC publishes Orphan Drug Development Guidebook
08/05/2020

UPDATE ON CLINICAL HOLD IGNITE DMD
07/05/2020

Caring for people with Duchenne, Becker and other MD's during COVID-19
01/05/2020

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