News Archive • World Duchenne

Showing all 30 results

Prof Aartsma-Rus separates the wheat from the scientific chaff. An overview of research papers for Duchenne & Becker MD in January 2021.

Monthly #apaperaday wrap-up: January 2022
28/01/2022

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.

#apaperaday: Repurposing of Medicines in the EU: Launch of a Pilot Framework
28/01/2022

A paper by Asker-Hagelberg et al from Frontiers In Medicine on the launch of the pilot framework for a medicine repurposing project.

Can symptomatic nmDuchenne carriers benefit from treatment with ataluren? Results of 193-month follow-up & Early treatment with Ataluren of a 2-year-old boy with nonsense mutation Duchenne dystrophy

#apaperaday: Can symptomatic nmDuchenne carriers benefit from treatment with ataluren? Results of 193-month follow-up & Early treatment with Ataluren of a 2-year-old boy with nonsense mutation Duchenne dystrophy
27/01/2022

Both case studies on ataluren. One on ataluren treatment for symptomatic carriers and one on ataluren treatment of a very young male patient.

#apaperaday: Diversity of Dystrophin Gene Mutations and Disease Progression in a Contemporary Cohort of Duchenne Muscular Dystrophy
26/01/2022

Paper on Duchenne mutations and their relationship to disease progression today. As expected there is a focus on heart pathology.

Morbidity, Clinical Course and Vaccination against SARS-CoV-2 Virus in Patients with Duchenne Muscular Dystrophy: A Patient Reported Survey

#apaperaday: Morbidity, Clinical Course and Vaccination against SARS-CoV-2 Virus in Patients with Duchenne Muscular Dystrophy: A Patient Reported Survey
21/01/2022

Today’s pick is from the international journal of environmental research and public health by Wasilewska et al on different aspects of COVID-19 in Duchenne patients in Poland. DOI 10.3390/ijerph19010406

#apaperaday: Human dystrophin expressing chimeric (DEC) cell therapy ameliorates cardiac, respiratory, and skeletal muscle's function in Duchenne muscular dystrophy
17/01/2022

Siemionow et al in Stem Cells Translational Medicine on stem cell transplantation of chimeric human myoblasts IN a MOUSE model.

ACT EU: Better clinical trials in the EU that address patients’ needs
13/01/2022

ACT EU: initiative to transform how clinical trials are initiated, designed and run, referred to as Accelerating Clinical Trials in the EU.

#apaperaday: Nonclinical data supporting orphan medicinal product designations: lessons from rare neurological conditions
11/01/2022

An older paper (2017) by Sheean et al from Drug Discovery Today on proper use of animal model data for orphan drug designation.

#apaperaday: Investigating the Potential for Sulforaphane to Attenuate Gastrointestinal Dysfunction in mdx Dystrophic Mice
10/01/2022

Today’s paper by Swiderski et al on testing if sulforaphane (SFN) can improve skeletal muscle and gastric muscle function in mdx mice.

#apaperaday: Pharmacological Profile of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy: A Japanese Experience
07/01/2022

Today’s a paper a day pick is from clinical pharmacology: advances and applications on viltolarsen by Roshmi and Prof. Toshi Yokota.

Edgewise announces results from Phase 1 EDG-5506 study
06/01/2022

Edgewise Therapeutics announced positive results from the Phase 1b portion of the first-in-human trial which enrolled individuals with Becker muscular dystrophy.

#apaperaday: Dystrophin deficiency affects human astrocyte properties and response to damage
04/01/2022

Today’s pick is by Lange et al from Glia. It is a study on astrocyte properties in cells with and without dystrophin.

#apaperaday: Alteration of skeletal and cardiac muscles function in DBA/2J mdx mice background: a focus on high intensity interval training
03/01/2022

Baati et al in Intractable and Rare Disease Research on the impact of high intensity training in d2/mdx mice.

2021 Wrap-up: Reflections, Activities, Moving Forward
22/12/2021

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
17/12/2021

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
15/12/2021

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
09/12/2021

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
24/11/2021

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
16/11/2021

VISION-DMD aims to develop a safe, effective and affordable therapy for Duchenne. This event presents the outcomes of the Vamorolone studies.

PPMD India joins World Duchenne Organization
09/11/2021

PPMD India joins World Duchenne Organization

New EU project BEAMER seeks to improve patient adherence to prescribed treatments
09/11/2021

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
05/11/2021

Invitation to participate in a short survey gathering opinions on returning clinical trial data to patients.

Warning statement on stem cell treatment

Warning on stem cell therapy in Duchenne
03/11/2021

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
18/10/2021

At Duchenne Patient Academy, DMD and BMD patient advocates receive training to build a strong base for current and future global advocacy.

STRIDE study data on Translarna ataluren

STRIDE data shows Translarna delays loss of ambulation by more than five years in nonsense DMD boys
23/09/2021

Data shows Translarna delays loss of ambulation by more than five years in nonsense DMD boys.

The Akari Foundation for Duchenne muscular dystrophy affected in Texas, United States

The Akari Foundation joins World Duchenne Organization
16/09/2021

The Akari Organization supports low-income Hispanic families with people affected by Duchenne in San Antonio, Texas.

ONLINE EVENT: ADULT LIFE & DUCHENNE
14/09/2021

The recording from the online event 'Adult Life & Duchenne' is now available for rewatching. Interpretation available in multiple languages.

World Duchenne Awareness Day highlights a year-round effort to improve adult life in Duchenne
10/09/2021

All World Duchenne Awareness Day 2021 events involving thousands of people across the world, under the theme ‘Adult Life & Duchenne’.

Consensus Guidelines for Adult Duchenne Care
08/09/2021

The Journal of Neuromuscular Diseases published the consensus guidelines for adult Duchenne care.

How Patient Organizations Can Drive FAIR Data Efforts to Facilitate Research and Health Care
01/09/2021

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.

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Monthly #apaperaday wrap-up: January 2022
28/01/2022

#apaperaday: Repurposing of Medicines in the EU: Launch of a Pilot Framework
28/01/2022

#apaperaday: Can symptomatic nmDuchenne carriers benefit from treatment with ataluren? Results of 193-month follow-up & Early treatment with Ataluren of a 2-year-old boy with nonsense mutation Duchenne dystrophy
27/01/2022

#apaperaday: Diversity of Dystrophin Gene Mutations and Disease Progression in a Contemporary Cohort of Duchenne Muscular Dystrophy
26/01/2022

#apaperaday: Morbidity, Clinical Course and Vaccination against SARS-CoV-2 Virus in Patients with Duchenne Muscular Dystrophy: A Patient Reported Survey
21/01/2022

#apaperaday: Human dystrophin expressing chimeric (DEC) cell therapy ameliorates cardiac, respiratory, and skeletal muscle's function in Duchenne muscular dystrophy
17/01/2022

ACT EU: Better clinical trials in the EU that address patients’ needs
13/01/2022

#apaperaday: Nonclinical data supporting orphan medicinal product designations: lessons from rare neurological conditions
11/01/2022

#apaperaday: Investigating the Potential for Sulforaphane to Attenuate Gastrointestinal Dysfunction in mdx Dystrophic Mice
10/01/2022

#apaperaday: Pharmacological Profile of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy: A Japanese Experience
07/01/2022

Edgewise announces results from Phase 1 EDG-5506 study
06/01/2022

#apaperaday: Dystrophin deficiency affects human astrocyte properties and response to damage
04/01/2022

#apaperaday: Alteration of skeletal and cardiac muscles function in DBA/2J mdx mice background: a focus on high intensity interval training
03/01/2022

2021 Wrap-up: Reflections, Activities, Moving Forward
22/12/2021

UN General Assembly Adopts first Resolution on people living with rare diseases
17/12/2021

Innovative and Sustainable FAIR solution for Duchenne Data Platform
15/12/2021

DMD community celebrates successful Duchenne Patient Academy 2021
09/12/2021

Topline results of Vamorolone after completing VISION-DMD
24/11/2021

VISION-DMD End Event: Vamorolone Clinical Trial Results
16/11/2021

PPMD India joins World Duchenne Organization
09/11/2021

New EU project BEAMER seeks to improve patient adherence to prescribed treatments
09/11/2021

Return of clinical trial data survey
05/11/2021

Warning on stem cell therapy in Duchenne
03/11/2021

Applications open for Duchenne Patient Academy 2021
18/10/2021

STRIDE data shows Translarna delays loss of ambulation by more than five years in nonsense DMD boys
23/09/2021

The Akari Foundation joins World Duchenne Organization
16/09/2021

ONLINE EVENT: ADULT LIFE & DUCHENNE
14/09/2021

World Duchenne Awareness Day highlights a year-round effort to improve adult life in Duchenne
10/09/2021

Consensus Guidelines for Adult Duchenne Care
08/09/2021

How Patient Organizations Can Drive FAIR Data Efforts to Facilitate Research and Health Care
01/09/2021

Warning on stem cell therapy in Duchenne

Adult Life & Duchenne

ENMC workshop to refine recommendations for female carriers