Translarna (ataluren) approved in Russia
PTC Therapeutics today announced that Translarna™ (ataluren) has been granted marketing approval in the Russian Federation for nonsense mutation Duchenne…
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Sarepta Announces Clinical Results from MOMENTUM study
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
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
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
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
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
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
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
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
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
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
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®
Today, Santhera announced the discontinuation of its Phase 3 SIDEROS study with Puldysa® (idebenone) in patients with Duchenne muscular dystrophy…
World Duchenne Awareness Day 2020 recap
Monday September 7 marked the 6th edition of World Duchenne Awareness Day. This international day dedicated to raise awareness about…
New Russian patient organization 'CF Gordey' joins WDO
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?
We all know that in Duchenne and Becker muscular dystrophy, the muscles are affected. However, there are also dystrophin variants…
Duchenne and the Brain
This conference has received funding from the European Union's Horizon 2020 research and innovation programme under grant agreement no…
ENMC workshop report: Brain dystrophin in muscular dystrophy
Thanks to the European NeuroMuscular Centre, last November a workshop on the role of brain dystrophin in muscular dystrophy; Implications…
Join the online program on Duchenne and the Brain
On September 7, we are launching a live online program for everyone interested in Duchenne care and research. Experts are…
Santhera tries to get worldwide rights to Vamorolone for all indications – including DMD
Santhera exercises the option to obtain worldwide rights of Vamorolone in Duchenne muscular dystrophy and all other indications. Santhera will…
Online program for brain involvement in Duchenne and Becker muscular dystrophy
An online program about brain involvement in Duchenne and Becker muscular dystrophy will launch on World Duchenne Awareness Day. This…
WDAD receives letter of support from Pope Francis
Just like previous years, Pope Francis is supporting the effort of World Duchenne Awareness Day to raise awareness for Duchenne…
FDA accepts review of casimersen for DMD patients amenable to skipping exon 45
Today, Sarepta announced the FDA accepted Casimersen for DMD patients amenable to skipping exon 45. The US Food and Drug Administration…
Official WDAD 2020 promo video launched
The Covid-19 pandemic has a strong impact on the life of every single individual and family, modifying our time and…
Exploring preclinical and clinical studies that might not have met expectation
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)
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
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
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
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
EURO-NMD, the European Reference Network for Rare Neuromuscular Diseases, spans 14 European countries, with 61 reference centres that oversee more…
