Monthly #apaperaday wrap-up: January 2022
Prof. Annemieke Aartsma-Rus is taking on a challenge by reading and commenting on a paper a day. See below the overview of January 2022.
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ACT EU: Better clinical trials in the EU that address patients’ needs
ACT EU: initiative to transform how clinical trials are initiated, designed and run, referred to as Accelerating Clinical Trials in the EU.
Edgewise announces results from Phase 1 EDG-5506 study
Edgewise Therapeutics announced positive results from the Phase 1b portion of the first-in-human trial which enrolled individuals with Becker muscular dystrophy.
2021 Wrap-up: Reflections, Activities, Moving Forward
The year 2022 is greeting us, bursting with new opportunities. Before waving goodbye to 2021, Elizabeth Vroom is reflecting back…
UN General Assembly Adopts first Resolution on people living with rare diseases
The UN General Assembly Adopts first Resolution on people living with rare diseases and their families.
Innovative and Sustainable FAIR solution for Duchenne Data Platform
The Duchenne Data Platform has successfully deployed an innovative and sustainable FAIR solution, achieving a FAIR status.
DMD community celebrates successful Duchenne Patient Academy 2021
Last week, the 5th edition of Duchenne Patient Academy has taken place virtually. More than 75 Duchenne and Becker MD patient advocates…
Topline results of Vamorolone after completing VISION-DMD
Vamorolone given throughout the study showed sustained efficacy across multiple endpoints over 48 weeks and the good safety and tolerability profile was confirmed.
VISION-DMD End Event: Vamorolone Clinical Trial Results
VISION-DMD aims to develop a safe, effective and affordable therapy for Duchenne. This event presents the outcomes of the Vamorolone studies.
New EU project BEAMER seeks to improve patient adherence to prescribed treatments
An international consortium will investigate factors affecting patient treatment adherence and develop a model to assess and understand patient needs,…
Return of clinical trial data survey
Invitation to participate in a short survey gathering opinions on returning clinical trial data to patients.
Warning on stem cell therapy in Duchenne
There are currently no approved stem cell therapies for DMD. You never pay for a clinical trial. Only trials on clinicaltrials.gov are legitimate.
Applications open for Duchenne Patient Academy 2021
At Duchenne Patient Academy, DMD and BMD patient advocates receive training to build a strong base for current and future global advocacy.
STRIDE data shows Translarna delays loss of ambulation by more than five years in nonsense DMD boys
Data shows Translarna delays loss of ambulation by more than five years in nonsense DMD boys.
The Akari Foundation joins World Duchenne Organization
The Akari Organization supports low-income Hispanic families with people affected by Duchenne in San Antonio, Texas.
ONLINE EVENT: ADULT LIFE & DUCHENNE
The recording from the online event 'Adult Life & Duchenne' is now available for rewatching. Interpretation available in multiple languages.
Adult Life & Duchenne online event
On 7 September 2021, the World Duchenne Organization hosted an online event on Adult Life & Duchenne. The event gathered…
World Duchenne Awareness Day highlights a year-round effort to improve adult life in Duchenne
All World Duchenne Awareness Day 2021 events involving thousands of people across the world, under the theme ‘Adult Life & Duchenne’.
World Duchenne Awareness Day highlights a year-round effort to improve adult life in Duchenne
On 7 September 2021, many activities around the world were organized on the occasion of World Duchenne Awareness Day. Awareness-raising…
Consensus Guidelines for Adult Duchenne Care
The Journal of Neuromuscular Diseases published the consensus guidelines for adult Duchenne care.
Consensus Guideline for Adult Duchenne Care
Today on World Duchenne Awareness Day, the Journal of Neuromuscular Diseases published the consensus guideline for adult Duchenne care. The…
A message from Pope Francis for World Duchenne Awareness Day
The World Duchenne Awareness Day is grateful to receive a message from Pope Francis. This year, he is once again…
How Patient Organizations Can Drive FAIR Data Efforts to Facilitate Research and Health Care
During the second FAIR Data Meeting for Duchenne, 120 participants from 22 countries discussed how they could drive FAIR data efforts to facilitate research and healthcare.
Adam Peska to win gold medal in Boccia at Tokyo Paralympics
Adam Peska from the Czech Republic won a gold medal in individual boccia BC3 at the 2020 Summer Paralympics in Tokyo, Japan.
#SPREADTHEWORD for World Duchenne Awareness Day
Today, the official World Duchenne Awareness Day 2021 promo video is launched. This video marks the start of the global…
Philip Jönsson first person with Duchenne MD to win gold at Paralympics
Today, Philip Jönsson won the gold medal in the mixed 10m air rifle standing SH2 event at the 2020 Summer Paralympics held in Tokyo, Japan.
ENMC workshop to refine recommendations for female carriers
The aim of this ENMC workshop were to raise priority and need for clinical and research focus on DMD female carriers. Report now available.
Online event on Adult Life & Duchenne
The World Duchenne Organization is organizing an online educational event on Adult Life & Duchenne.
Online event on Adult Life & Duchenne
The World Duchenne Organization is organizing an online educational event on Adult Life & Duchenne. The free event on September…
