September 18, 2017September 18, 2017
‘Coach to Cure MD’ and Other Fundraising Events Mark Duchenne Muscular Dystrophy Month
News
For the ninth year in a row, “Coach to Cure MD” and Parent Project Muscular Dystrophy (PPMD) will mark Duchenne Awareness Month by ... Read more
September 15, 2017
Santhera to Appeal European Agency’s Decision Against Raxone as a Duchenne MD Therapy
News
The agency that plays the key role in European Union therapy authorizations has recommended against approval of Raxone (idebenone) as a Duchenne ... Read more
September 14, 2017September 13, 2017
Tips and Tricks to Stay Positive when You Have a Disability
columns, You, Me and MD - a Column by Leah Leilani
It’s not always easy to be positive with a disability, especially in the beginning. It’s hard to see your ... Read more
September 14, 2017September 16, 2017
Jerry Mendell, in PPMD Webinar, Details Plans for Possible Trial of Duchenne Gene Therapy
News
In a recent webinar, Dr. Jerry Mendell with Nationwide Children’s Hospital rolled out plans for a Phase 1/2a clinical trial ... Read more
September 13, 2017September 13, 2017
Shortened Telomeres Linked to Muscle Degeneration in Duchenne, Study Finds
News
Young muscle stem cells in patients with Duchenne muscular dystrophy have shortened telomeres, causing these cells to be less able ... Read more
September 12, 2017
Utah Researcher Receives Grant to Study Stages of Congenital Myotonic Dystrophy
News
A University of Utah medical professor has received a $600,000 Muscular Dystrophy Association grant to conduct a clinical trial aimed at understanding how congenital ... Read more
September 12, 2017September 19, 2017
First DMD Patients to Receive Raxone Under U.K.’s Early Access Program
News
The first patients with Duchenne muscular dystrophy (DMD) have been enrolled into the U.K.’s Early Access to Medicines Scheme ... Read more
September 11, 2017September 11, 2017
Sarepta’s Exon 53 Skipping Therapy, Golodirsen, Improves Dystrophin Expression in Phase 1/2 Trial
News
Sarepta Therapeutics says its lead candidate therapy for exon 53 skipping, golodirsen, showed potential to treat Duchenne muscular dystrophy (DMD) in ... Read more
September 8, 2017
On World Duchenne Awareness Day, Winners of 2017 STRIVE Awards Announced
News
PTC Therapeutics recently announced the recipients of the 2017 global Strategies to Realize Innovation, Vision and Empowerment (STRIVE) Awards program, ... Read more
September 7, 2017
Parent Project Muscular Dystrophy Holding Duchenne Action Month During September
News
Parent Project Muscular Dystrophy is holding an awareness, fund-raising and educational campaign throughout September to fight Duchenne muscular dystrophy (DMD). ... Read more
September 6, 2017
Gene Therapy for OPMD Nears Human Studies, Benitec Announces
News
A single gene therapy that silences the mutation responsible for oculopharyngeal muscular dystrophy (OPMD) and replaces the mutated gene ... Read more
September 5, 2017
Upcoming Duchenne Gene Therapy Trial to Be Focus of PPMD-hosted Webinar on Wednesday, Sept. 6
News
Parent Project Muscular Dystrophy (PPMD) will host a one-hour webinar at 2 p.m. Eastern Time on Wednesday, Sept. 6, that will ... Read more

‘Coach to Cure MD’ and Other Fundraising Events Mark Duchenne Muscular Dystrophy Month

For the ninth year in a row, “Coach to Cure MD” and Parent Project Muscular Dystrophy (PPMD) will mark Duchenne Awareness Month by using college football games to raise awareness of Duchenne muscular ... Read more

September 15, 2017

Santhera to Appeal European Agency’s Decision Against Raxone as a Duchenne MD Therapy

News

The agency that plays the key role in European Union therapy authorizations has recommended against approval of Raxone (idebenone) as a Duchenne muscular dystrophy (DMD) treatment. Santhera said in a press release that it plans to ... Read more

September 14, 2017September 13, 2017

Tips and Tricks to Stay Positive when You Have a Disability

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

It’s not always easy to be positive with a disability, especially in the beginning. It’s hard to see your friends do things that you can’t. The stares are hard ... Read more

September 14, 2017September 16, 2017

Jerry Mendell, in PPMD Webinar, Details Plans for Possible Trial of Duchenne Gene Therapy

News

In a recent webinar, Dr. Jerry Mendell with Nationwide Children’s Hospital rolled out plans for a Phase 1/2a clinical trial of a new gene therapy for Duchenne’s muscular dystrophy (DMD). ... Read more

September 13, 2017September 13, 2017

Shortened Telomeres Linked to Muscle Degeneration in Duchenne, Study Finds

News

Young muscle stem cells in patients with Duchenne muscular dystrophy have shortened telomeres, causing these cells to be less able to build new muscle, according to new research. A full ... Read more

September 12, 2017

Utah Researcher Receives Grant to Study Stages of Congenital Myotonic Dystrophy

News

A University of Utah medical professor has received a $600,000 Muscular Dystrophy Association grant to conduct a clinical trial aimed at understanding how congenital myotonic dystrophy develops over time. The three-year grant will allow Dr. Nicholas ... Read more

September 12, 2017September 19, 2017

First DMD Patients to Receive Raxone Under U.K.’s Early Access Program

News

The first patients with Duchenne muscular dystrophy (DMD) have been enrolled into the U.K.’s Early Access to Medicines Scheme (EAMS) for Santhera Pharmaceuticals’ Raxone (idebenone), giving patients access to ... Read more

September 11, 2017September 11, 2017

Sarepta’s Exon 53 Skipping Therapy, Golodirsen, Improves Dystrophin Expression in Phase 1/2 Trial

News

Sarepta Therapeutics says its lead candidate therapy for exon 53 skipping, golodirsen, showed potential to treat Duchenne muscular dystrophy (DMD) in a first clinical trial of DMD patients. According to results of ... Read more

September 8, 2017

On World Duchenne Awareness Day, Winners of 2017 STRIVE Awards Announced

News

PTC Therapeutics recently announced the recipients of the 2017 global Strategies to Realize Innovation, Vision and Empowerment (STRIVE) Awards program, recognizing excellence and innovation in the Duchenne muscular dystrophy (DMD) community. ... Read more

September 7, 2017

Parent Project Muscular Dystrophy Holding Duchenne Action Month During September

News

Parent Project Muscular Dystrophy is holding an awareness, fund-raising and educational campaign throughout September to fight Duchenne muscular dystrophy (DMD). It has scheduled events every day of Duchenne Action Month. You’ll ... Read more

September 6, 2017

Gene Therapy for OPMD Nears Human Studies, Benitec Announces

News

A single gene therapy that silences the mutation responsible for oculopharyngeal muscular dystrophy (OPMD) and replaces the mutated gene with a normal one may advance into human studies in ... Read more

September 5, 2017

Upcoming Duchenne Gene Therapy Trial to Be Focus of PPMD-hosted Webinar on Wednesday, Sept. 6

News

Parent Project Muscular Dystrophy (PPMD) will host a one-hour webinar at 2 p.m. Eastern Time on Wednesday, Sept. 6, that will focus on an upcoming clinical trial exploring gene therapy for ... Read more