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…
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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…
#WDAD2020: Together we are stronger
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
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
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
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
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
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
Today, Nippon Shinyaku announced that the European Commission (EC) has granted Orphan Drug Designation to viltolarsen. Viltolarsen is an antisense…
2020: Duchenne and the Brain
Duchenne and the Brain September 7 is World Duchenne Awareness Day. On this day we raise awareness for Duchenne and…
Regulatory Review of Santhera's Idebenone by the CHMP
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
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
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)
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…
