News Archive - World Duchenne

Showing all 30 results

Will the COVID-19 vaccine impact my possibility to have gene therapy?
12/01/2021

Gene therapy uses viruses to deliver a micro-dystrophin gene or an exon skipping gene. COVID-19 is a virus. If I…

Lay summaries of SPITFIRE and THUNDERJET studies available
11/01/2021

All results of clinical trials generate new knowledge, and helps to advance the development of treatments. This includes the sharing…

Translarna (ataluren) approved in Russia
08/12/2020

PTC Therapeutics today announced that Translarna™ (ataluren) has been granted marketing approval in the Russian Federation for nonsense mutation Duchenne…

Sarepta Announces Clinical Results from MOMENTUM study
07/12/2020

Today, Sarepta shared an update for its investigational PPMO SRP-5051 clinical trial study. MOMENTUM, the name of their phase 2…

Genethon gets approval to start gene therapy trial
04/12/2020

Genethon is dedicated to design and develop a gene therapy products for rare diseases. On Monday 30th of November it…

The experience as mother of a Duchenne adult who recently turned 30
19/11/2020

Elizabeth Vroom shares her experience as mother of a Duchenne young man who recently celebrated his 30th birthday. Elizabeth Vroom…

International congress on the effects of COVID-19 on people with rare diseases
05/11/2020

On the second week of November, an international conference will take place virtually in order to present the patient registry…

Enlace Mexico: WDO’s 45th Member Organization
03/11/2020

Enlace Distrofia Muscular Duchenne Becker is the only non-profit organization providing optimal medical, emotional and psychological care for DMD/BMD patients…

Report: World Muscle Society (WMS) 2020 International Congress
27/10/2020

This year the 25th edition of the World Muscle Society (WMS) 2020 International Congress, a congress where the worldwide important…

WDO welcomes 44th member patient organization: DMD Aileleri
27/10/2020

DMD Aileleri Derneği (meaning DMD Families Association in Turkish) started Turkey by families whose children are diagnosed with Duchenne and…

Phase 3 PolarisDMD trial with edasalonexent stopped
27/10/2020

Our hearts are heavy when the news reached us that the Phase 3 PolarisDMD trial with edasalonexent did not meet…

FDA Orphan Drug Designation for ATL1102 for DMD
26/10/2020

Antisense Therapeutics announced that the US Food and Drug Administration (FDA) Office of Orphan Products Development has granted an Orphan…

Duchenne Patient Academy 2020: Applications now open
26/10/2020

We are delighted to announce that Duchenne Patient Academy, supported by the World Duchenne Organization, is returning for the fourth…

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…

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Will the COVID-19 vaccine impact my possibility to have gene therapy?
12/01/2021

Lay summaries of SPITFIRE and THUNDERJET studies available
11/01/2021

Translarna (ataluren) approved in Russia
08/12/2020

Sarepta Announces Clinical Results from MOMENTUM study
07/12/2020

Genethon gets approval to start gene therapy trial
04/12/2020

The experience as mother of a Duchenne adult who recently turned 30
19/11/2020

International congress on the effects of COVID-19 on people with rare diseases
05/11/2020

Enlace Mexico: WDO’s 45th Member Organization
03/11/2020

Report: World Muscle Society (WMS) 2020 International Congress
27/10/2020

WDO welcomes 44th member patient organization: DMD Aileleri
27/10/2020

Phase 3 PolarisDMD trial with edasalonexent stopped
27/10/2020

FDA Orphan Drug Designation for ATL1102 for DMD
26/10/2020

Duchenne Patient Academy 2020: Applications now open
26/10/2020

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

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

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

The experience as mother of a Duchenne adult who recently turned 30