News

Elizabeth Vroom shares her experience as mother of a Duchenne young man who recently celebrated his 30th birthday. Elizabeth Vroom is the mother of Justus, a 30-year-old adult living with Duchenne muscular dystrophy. Next to this she is also a prominent figure in the international Duchenne Community as Chair of the World Duchenne Organization. Duchenne […]

The post The experience as mother of a Duchenne adult who recently turned 30 appeared first on World Duchenne.

On the second week of November, an international conference will take place virtually in order to present the patient registry of COVID-19 and rare diseases and to discuss diverse topics of interest related to the current pandemic. The International Congress on the Effects of COVID-19 on People with Rare Diseases will be held on November […]

The post International congress on the effects of COVID-19 on people with rare diseases appeared first on World Duchenne.

Enlace Distrofia Muscular Duchenne Becker is the only non-profit organization providing optimal medical, emotional and psychological care for DMD/BMD patients and their families in Northern Mexico. Since 2000, the organization supports families in the states of Chihuahua and Nuevo León. For the last twenty years, Enlace has been developing their learnings into a model to […]

The post Enlace Mexico: WDO’s 45th Member Organization appeared first on World Duchenne.

This year the 25th edition of the World Muscle Society (WMS) 2020 International Congress, a congress where the worldwide important updates of research on Duchenne Muscular Dystrophies (DMD) and Becker (BMD) are shown, was held virtually. Although there were many posters presented, Dr. Marisol Montolio has created short summary of the most important highlights for […]

The post Report: World Muscle Society (WMS) 2020 International Congress appeared first on World Duchenne.

DMD Aileleri Derneği (meaning DMD Families Association in Turkish) started Turkey by families whose children are diagnosed with Duchenne and Becker Muscular Dystrophy. The combined shape of DNA chain and sandglass in the organization’s logo represents the race against time on this genetic disease. They have a clear vision: “Improve the standards of living for […]

The post WDO welcomes 44th member patient organization: DMD Aileleri appeared first on World Duchenne.

Our hearts are heavy when the news reached us that the Phase 3 PolarisDMD trial with edasalonexent did not meet its primary or secondary endpoints. Families involved in the PolarisDMD and GalaxyDMD trials who have questions are encouraged to contact their study site directly. All in the Duchenne community are welcome to join the webinar […]

The post Phase 3 PolarisDMD trial with edasalonexent stopped appeared first on World Duchenne.

Antisense Therapeutics announced that the US Food and Drug Administration (FDA) Office of Orphan Products Development has granted an Orphan Drug Designation (ODD) to ATL1102 for treatment of Duchenne muscular dystrophy (DMD). ATL1102 is an antisense drug designed to reduce inflammation in human disease. Read the full press release here (external link) It was recently […]

The post FDA Orphan Drug Designation for ATL1102 for DMD appeared first on World Duchenne.

We are delighted to announce that Duchenne Patient Academy, supported by the World Duchenne Organization, is returning for the fourth year this November. Applications are now open! Patient advocates looking for training in the field of research, policy, regulations and data are highly encouraged to apply. The online program takes place over the week from […]

The post Duchenne Patient Academy 2020: Applications now open appeared first on World Duchenne.

Living with Duchenne and Becker muscular dystrophy (DMD and BMD) has a profound impact on the lives and mental health of the individual and their family. People affected may suffer from depression, stress and anxiety. These crucial aspects of the disease are often neglected. Especially during the current COVID-19 pandemic, causing additional stress and anxiety, […]

The post Attention to mental well-being in DMD/BMD families appeared first on World Duchenne.

Today, Santhera announced the discontinuation of its Phase 3 SIDEROS study with Puldysa® (idebenone) in patients with Duchenne muscular dystrophy (DMD) who are in respiratory decline and receive concomitant glucocorticoid treatment. Data from an interim analysis conducted by the independent Data and Safety Monitoring Board (DSMB) concluded that the study was unlikely to meet its […]

The post Santhera stops Phase 3 SIDEROS Study and Development of Puldysa® appeared first on World Duchenne.

PPMD is excited to welcome Lilly back to the Duchenne space! With Lilly’s experience in Duchenne, we are thrilled to see their return to the field and look forward to learning more as this early...

The post Lilly and Precision BioSciences Announce Genome Editing Research Collaboration and License Agreement appeared first on Parent Project Muscular Dystrophy.

Join us on Sunday, November 22 at 7 PM EST for an incredible evening of music on the PPMD Facebook Page! PPMD’s Community Concert: Harmony Together will feature members of the Duchenne community sharing their...

The post Attend PPMD’s Community Concert: Harmony Together appeared first on Parent Project Muscular Dystrophy.

PPMD has launched our second COVID-19 Pandemic Impact Survey related to your experiences during the pandemic for accessing care, trials, and approved therapies. Thank you to everyone who completed our first survey earlier in the spring....

The post NEW – Fall 2020 Survey: Impact of the COVID-19 Pandemic on Duchenne appeared first on Parent Project Muscular Dystrophy.

Another historic year for Sam’s Night! Last week, the Killian Family and friends joined “together” to celebrate PPMD’s longest running signature DIY event. With $506,294 raised, 2020 marked the 15th Anniversary, the first virtual edition,...

The post 15th Annual Sam’s Night Raises Over $500,000 in the Fight to End Duchenne appeared first on Parent Project Muscular Dystrophy.

Shop for gifts this holiday season with AmazonSmile or while rounding up your spare change using the Cheerful app and generate donations for PPMD. Round Up your Spare Change Round up on your purchases to...

The post Shop Throughout the Holiday Season and Support PPPMD at the Same Time appeared first on Parent Project Muscular Dystrophy.

The National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH) has awarded a $3.3 million grant to ReveraGen Inc. to support the advancement of vamorolone towards FDA submission and...

The post ReveraGen Receives $3.3 Million NIH Grant for NDA Preparations for Vamorolone in Duchenne appeared first on Parent Project Muscular Dystrophy.

In an unprecedented collaboration designed to speed research advances, Duchenne UK, the Muscular Dystrophy Association (MDA), and Parent Project Muscular Dystrophy (PPMD) have announced a joint grant of $686,500 to Dr. William Evans (principal investigator)...

The post Duchenne UK, MDA & PPMD Award $686,500 Grant To Develop Easier Way To Measure if New Treatments Are Working appeared first on Parent Project Muscular Dystrophy.

We are just days away from the 13th Annual Coach To Cure MD presented by Werner Ladder on Saturday, November 14! This weekend, AFCA Coaches around the country will be wearing Coach To Cure MD patches,...

The post Get Ready to Tackle Duchenne! Coach To Cure MD is this Weekend! appeared first on Parent Project Muscular Dystrophy.

14,000 Babies Tested in Partnership with New York State and NBSTRN; Data Presented at Association of Public Health Laboratories Newborn Screening Virtual Symposium Parent Project Muscular Dystrophy (PPMD) today announced that our Newborn Screening Pilot...

The post PPMD’s Ground-Breaking Effort: An Update After One Year of Our Newborn Screening Pilot for Duchenne appeared first on Parent Project Muscular Dystrophy.

Fall is here and college football is in full swing…which means it is time to huddle up for the 13th Annual Coach To Cure MD, presented by Werner Ladder on Saturday, November 14! As we all...

The post Exciting news! Country star Chase Rice joins Coach To Cure MD! appeared first on Parent Project Muscular Dystrophy.