2022 Wrap-up: Activities of World Duchenne Organization
Elizabeth Vroom looks back on 2022, listing all activities of the World Duchenne Organization, and carefully draws a rough outline for 2023.
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Monthly #apaperaday wrap-up: November 2022
Prof. Annemieke Aartsma-Rus is taking on a challenge by reading and commenting on a paper a day. See below the overview of November 2022.
MENA Organization for Rare Diseases Annual Meeting
The MENA Organization for Rare Diseases Annual Meeting is held in Dubai from March 3-5, 2023. The region's largest rare diseases event.
Webinar: Patient involvement in the development, regulation and safe use of medicines
January 31, the European Medicines Agency hosts a webinar on patient involvement in the development, regulation and safe use of medicines.
Duchenne Patient Academy 2022 successfully concluded
Duchenne Patient Academy 2022 provides training and education opportunities for people involved in Duchenne and Becker MD patient advocacy.
Dr Imelda de Groot award for improving care in Duchenne
The ‘Dr Imelda de Groot’ is an annual award and meant to stimulate persons to improve care for persons with Duchenne muscular dystrophy (DMD).
Monthly #apaperaday wrap-up: October 2022
Prof. Annemieke Aartsma-Rus is taking on a challenge by reading and commenting on a paper a day. See below the overview of October 2022.
Santhera and ReveraGen Complete NDA Submission to FDA and MAA Submission to EMA for Vamorolone
Santhera and ReveraGen Complete NDA Submission to FDA and MAA Submission for EMA for Vamorolone as a potential treatment for DMD.
Submit a Poster for Duchenne Patient Academy 2022
Do you have an innovative project related to the Duchenne field? Submit an E-Poster for Duchenne Patient Academy 2022!
Meeting on FAIR Data ‘Visiting’ for Duchenne and other Rare Diseases
The World Duchenne Organization is pleased to announce that the 3rd International FAIR data ‘visiting’ for Duchenne & Other Rare…
Advisory Board for the ADC Program fully installed
Today, the Advisory Board of the ADC Program is fully installed. We are incredibly proud and honored to have these…
Duchenne Patient Academy 2022 Applications Now Open
Duchenne Patient Academy 2022 Applications Now Open. Help drive the international advocacy agenda for people living with dystrophinopathies.
World Duchenne Organization announces the Accredited Duchenne Centers Program
Amsterdam, 4 October 2022 – The World Duchenne Organization is pleased to announce the launch of the Accredited Duchenne Centers…
World Duchenne Organization announces Accredited Duchenne Centers Program
World Duchenne Organization announces the launch of the Accredited Duchenne Centers Program, a worldwide accreditation for Duchenne Centers.
ENMC Workshop: Focus on female carriers of dystrophinopathy
13-15 May 2022, an ENMC workshop was organized on females carriers of dystrophinopathy. The lay summary is available in several languages.
Webinar: Información del Estudio BIND para Familias en España
Un seminario web para familias en España para el estudio BIND sobre desafíos de aprendizaje y comportamiento en Duchenne y Becker MD
Patient involvement in the development, regulation and safe use of medicines
New CHIOMS report on patient involvement in the development, regulation and safe use of medicines is now available for download.
#apaperaday special: PPMD meeting report on females with dystrophinopathy
Parent Project Muscular Dystrophy Females with Dystrophinopathy Conference, Orlando, Florida June 26 – June 27, 2019
World Duchenne Organization Leadership Award for Professor Muntoni
WDO Leadership Award, the highest recognition for individual achievement, is awarded to Professor Francesco Muntoni.
#apaperaday special: How much dystrophin is needed for survival and function
Today’s pick is one of my own papers by van Putten et al in FASEB journal doi 10.1096/fj.12-224170. Here we…
#apaperaday special: How much dystrophin is needed for survival and function
How much dystrophin is needed for survival and function in Duchenne? Paper shows that little bits of dystrophin already can go a long way.
FAIR Data Sharing Without Oversharing
Duchenne Parent Project NL and EURO-NMD can now begin leveraging their FAIR data to benefit People Living With Rare Diseases.
#apaperaday special: Needs of mothers caring for children with Duchenne
Today’s pick for the Women and Duchenne theme of World Duchenne Awareness Day focuses on female caregivers (usually mothers) of…
#apaperaday special: Needs of mothers caring for children with Duchenne
Today’s pick for the Women and Duchenne theme focuses on female caregivers (usually mothers) of Duchenne patients and their needs.
#apaperaday special: Why some Duchenne carriers have symptoms
Today's Women and Duchenne paper tries to elucidate why some female Duchenne carriers have symptoms and others have not.
#apaperaday special: Why some Duchenne carriers have symptoms
Women and Duchenne theme continued with another paper trying to elucidate why some female carriers have symptoms and others have…
#apaperaday special: Cause of manifesting Duchenne carriers
Dilated cardiomyopathy is frequent in manifesting Duchenne carriers. Also, women can be a carrier if they don't have a relative with Duchenne.
#apaperaday special: Cause of manifesting Duchenne carriers
Today and tomorrow we dive into the cause of manifesting carriers starting with a paper by Soltanzadeh et al in…
#apaperaday special: Cardiac Care for Female Carriers of Duchenne
Today’s pick in #apaperaday “Women and Duchenne” special is a letter to the editor by Wang et al in the…
#apaperaday special: Cardiac Care for Female Carriers of Duchenne
Cardiac Care for Female Carriers of Duchenne: 2/3 mothers with a child with Duchenne is at risk of developing dilated cardiomyopathy.
