News

Nominations for the EURORDIS Black Pearl Award 2022 are now open!

The post EURORDIS Black Pearl Awards 2022 appeared first on World Duchenne.

2. Adult Life & Duchenne
13 July 2021, 11:43 am

On September 7, the World Duchenne Organization will be hosting an online event where experts share their stories and experiences surrounding DMD/BMD and adult life.

The post Adult Life & Duchenne appeared first on World Duchenne.

From October 26 - 29, the European Patients’ Forum (EPF) is organizing a congress on the digital transformation of healthcare.

The post EPF Congress 2021: Digital Transformation of Healthcare appeared first on World Duchenne.

Today is the First International Neonatal Screening Day. Early diagnosis is of great importance for everybody with medical problems.

The post First International Neonatal Screening Day appeared first on World Duchenne.

During the Duchenne Care Conference, top experts share their experience on specific topics of Duchenne (and Becker) care.

The post Duchenne Care Conference 2021 successfully concluded appeared first on World Duchenne.

Duchenne Ukraine opens an international children's drawing competition to discover and celebrate talented children living with Duchenne.

The post Duchenne Ukraine announces international children’s drawing competition appeared first on World Duchenne.

September 7 is World Duchenne Awareness Day. This year's theme that deserves more attention is ‘Adult Life & Duchenne’.

The post World Duchenne Awareness Day 2021 appeared first on World Duchenne.

8. Dr Imelda de Groot Award
3 June 2021, 7:36 pm

During the Duchenne Care Conference, taking place June 1-4 online, the Dr Imelda de Groot Award was announced. The Award was revealed by Elizabeth Vroom, chair of the World Duchenne Organization.

The post Dr Imelda de Groot Award appeared first on World Duchenne.

Share4Rare shares the findings and outputs of their project, setting the base for the next generation of data sharing in rare diseases.

The post Share4Rare: Setting the path for next generation data sharing in rare diseases appeared first on World Duchenne.

DMD Care Association provides support for children and families affected by Duchenne in Romania. They are World Duchenne Organization member.

The post DMD Care Association Romania joins World Duchenne Organization appeared first on World Duchenne.

PPMD takes every action necessary to ensure therapies are developed efficiently and that they include the voice of the community. In 2014, we came together as a community, creating and submitting to the FDA the...

The post PPMD Begins Process to Update Community-Led Duchenne Guidance for FDA appeared first on Parent Project Muscular Dystrophy.

We need your help! Please encourage your Senators to cosponsor the BENEFIT Act. This legislation will amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, PFDD and related data – including...

The post Action Alert: Urge your House and Senate Members to Cosponsor the BENEFIT Act appeared first on Parent Project Muscular Dystrophy.

PPMD applauds Representatives Matsui and Wenstrup for today’s introduction of the bipartisan, commonsense BENEFIT Act, legislation conceived by PPMD that will help ensure the patient voice is at the center of the FDA’s review process....

The post Reps. Matsui & Wenstrup Introduce the Bipartisan BENEFIT Act to Increase Patients’ Role in Drug Approvals Process appeared first on Parent Project Muscular Dystrophy.

Teens & Adults with Duchenne & Becker: Join the PAAC Chats App PPMD’s Adult Advisory Committee, better known as the PAAC, has launched a new community platform for teens and adults living with Duchenne and...

The post New! Teens & Adults: Join the PAAC Chats App appeared first on Parent Project Muscular Dystrophy.

This year marks 20 years since passage of the MD-CARE Act, landmark legislation led by PPMD that ushered in a new era of federal investment into the muscular dystrophies. This year also marked the first...

The post 122 Members of Congress Send Letter of Strong Support for Duchenne Priorities & Funding for 2022 Budget appeared first on Parent Project Muscular Dystrophy.

PPMD’s 2021 Virtual Annual Conference: Recently Reported Updates As a new addition to PPMD’s 2021 Virtual Conference, PPMD invited industry partners to share new data with the community live during the Conference in a series...

The post Recently Reported Updates from PPMD’s 2021 Virtual Annual Conference appeared first on Parent Project Muscular Dystrophy.

PPMD’s Main Office is Moving to Washington, DC! When Parent Project Muscular Dystrophy started in 1994, formed by a small group of parents and grandparents out of Ohio, we knew that one of our first...

The post PPMD Is Moving to Washington, DC! appeared first on Parent Project Muscular Dystrophy.

Did You Miss PPMD’s 2021 Virtual Annual Conference? PPMD’s 2021 Virtual Annual Conference was incredible with close to 2,000 families, care providers, researchers, scientists, and industry partners joining us for four days of informative updates...

The post Rewatch PPMD’s 2021 Virtual Annual Conference appeared first on Parent Project Muscular Dystrophy.

This afternoon at PPMD’s Virtual Annual Conference, Italfarmaco shared Phase II Study results from the Givinostat trial in patients with Becker. They also issued a press release below. Although they did not meet the primary...

The post Italfarmaco Announces Topline Data from Phase 2 Trial with Givinostat in Patients with Becker appeared first on Parent Project Muscular Dystrophy.

PPMD’s 2021 Virtual Annual Conference to Feature Five Late-Breaking News Sessions We are excited to share that five Industry Partners will be announcing newly released data to the Duchenne community for the first time during...

The post PPMD Virtual Conference to Feature 5 Late-Breaking News Sessions appeared first on Parent Project Muscular Dystrophy.