September 24, 2018September 24, 2018
College Football Coaches Joining Fight Against DMD During Annual Coach To Cure MD Event
News
On Saturday, coaches with the American Football Coaches Association (AFCA) across the U.S. will display patches to promote the annual Coach ... Read more
September 21, 2018
#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington
News
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next ... Read more
September 21, 2018September 21, 2018
Nonprofit Coalition to Cure Calpain 3 Funds 2 Gene Therapy Research Projects for LGMD2A
News
Two new research projects have been funded by the Coalition to Cure Calpain 3 (C3), a nonprofit patient advocacy ... Read more
September 19, 2018September 19, 2018
Potential DMD Therapy Vamorolone Shows Positive Effects in Phase 2a Trial, Study Reports
News
Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid ... Read more
September 18, 2018September 17, 2018
A Girl’s Best Friend: Thoughts on Mobility Assistance Dogs
columns, You, Me and MD - a Column by Leah Leilani
One of the best days of my life was when my family and I brought home my dog, Andre. ... Read more
September 17, 2018September 17, 2018
Obsessive Compulsive Disorders Evident in Duchenne Patients and in Need of Treatment, Study Says
News
Obsessive compulsive disorder (OCD), a type of “internalizing disorder,” are evident in children with Duchenne muscular dystrophy, and particularly associated ... Read more
September 14, 2018September 14, 2018
Gene Editing Restored Dystrophin Levels in Dog Model of Duchenne MD, Study Reports
News
Using the gene editing process CRISPR/Cas9 enabled a marked restoration of dystrophin production in various muscles of dogs with ... Read more
September 12, 2018September 12, 2018
STRIVE Awards Recognize Efforts of Duchenne MD Patient Groups Worldwide
News
PTC Therapeutics announced the winners of the 2018 global Strategies to Realize Innovation, Vision and Empowerment (STRIVE) awards, which ... Read more
September 11, 2018September 7, 2018
Listening and Surrendering to My Body Isn’t Always Easy
columns, You, Me and MD - a Column by Leah Leilani
Being a healthy able-bodied human being must be nice. Not having to worry about taking medications or planning out ... Read more
September 10, 2018September 10, 2018
Numotion, Sing Me a Story Partner to Help Children with Neuromuscular Diseases Turn Life Stories to Songs
News
Nonprofit Sing Me a Story and Numotion are collaborating to help children with various neuromuscular diseases served by the Muscular Dystrophy ... Read more
September 7, 2018September 7, 2018
Pfizer Stops Phase 2 Clinical Studies of Domagrozumab in DMD Boys
News
Pfizer is halting the clinical development of domagrozumab (PF-06252616) as treatment for Duchenne muscular dystrophy (DMD) in an ... Read more
September 5, 2018September 5, 2018
PPMD and Jett Foundation Among Groups Gearing Up for Sept. 7, World Duchenne Awareness Day
News
With an emphasis on standards of care, groups such as the Parent Project Muscular Dystrophy and the Jett Foundation ... Read more

College Football Coaches Joining Fight Against DMD During Annual Coach To Cure MD Event

News

On Saturday, coaches with the American Football Coaches Association (AFCA) across the U.S. will display patches to promote the annual Coach To Cure MD initiative during college football games. The fundraising effort, ... Read more

September 21, 2018

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

News

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products ... Read more

September 21, 2018September 21, 2018

Nonprofit Coalition to Cure Calpain 3 Funds 2 Gene Therapy Research Projects for LGMD2A

News

Two new research projects have been funded by the Coalition to Cure Calpain 3 (C3), a nonprofit patient advocacy organization, to develop gene therapies for limb-girdle muscular dystrophy type ... Read more

September 19, 2018September 19, 2018

Potential DMD Therapy Vamorolone Shows Positive Effects in Phase 2a Trial, Study Reports

News

Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid prednisolone in a Phase 2a clinical trial for boys 4 ... Read more

September 18, 2018September 17, 2018

A Girl’s Best Friend: Thoughts on Mobility Assistance Dogs

columns, You, Me and MD - a Column by Leah Leilani

One of the best days of my life was when my family and I brought home my dog, Andre. The second my eyes fell on him, I melted into ... Read more

September 17, 2018September 17, 2018

Obsessive Compulsive Disorders Evident in Duchenne Patients and in Need of Treatment, Study Says

News

Obsessive compulsive disorder (OCD), a type of “internalizing disorder,” are evident in children with Duchenne muscular dystrophy, and particularly associated with anxiety and places considerable stress on the patient’s family, ... Read more

September 14, 2018September 14, 2018

Gene Editing Restored Dystrophin Levels in Dog Model of Duchenne MD, Study Reports

News

Using the gene editing process CRISPR/Cas9 enabled a marked restoration of dystrophin production in various muscles of dogs with Duchenne muscular dystrophy (DMD). The study with that finding, “Gene editing ... Read more

September 12, 2018September 12, 2018

STRIVE Awards Recognize Efforts of Duchenne MD Patient Groups Worldwide

News

PTC Therapeutics announced the winners of the 2018 global Strategies to Realize Innovation, Vision and Empowerment (STRIVE) awards, which recognize the efforts of nonprofit organizations working to serve the ... Read more

September 11, 2018September 7, 2018

Listening and Surrendering to My Body Isn’t Always Easy

columns, You, Me and MD - a Column by Leah Leilani

Being a healthy able-bodied human being must be nice. Not having to worry about taking medications or planning out your day based on your energy levels. Not having to ... Read more

September 10, 2018September 10, 2018

Numotion, Sing Me a Story Partner to Help Children with Neuromuscular Diseases Turn Life Stories to Songs

News

Nonprofit Sing Me a Story and Numotion are collaborating to help children with various neuromuscular diseases served by the Muscular Dystrophy Association (MDA) share their unique stories through song. This alliance ... Read more

September 7, 2018September 7, 2018

Pfizer Stops Phase 2 Clinical Studies of Domagrozumab in DMD Boys

News

Pfizer is halting the clinical development of domagrozumab (PF-06252616) as treatment for Duchenne muscular dystrophy (DMD) in an ongoing Phase 2 trial. The latest trial data failed to demonstrate ... Read more

September 5, 2018September 5, 2018

PPMD and Jett Foundation Among Groups Gearing Up for Sept. 7, World Duchenne Awareness Day

News

With an emphasis on standards of care, groups such as the Parent Project Muscular Dystrophy and the Jett Foundation are gearing up for Sept. 7, World Duchenne Awareness Day ... Read more