Entries by Marie-Catherine Du Berger

Raising my son with Duchenne

Living with Duchenne muscular dystrophy is difficult. I understand it because I live this reality every day. Duchenne muscular dystrophy forces you to live the moment and enjoy it even more. Christine Winslow from Johannesburg South Africa shared her story on the Facebook page of her son, Jason, who lives with DMD. She wants this […]

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News about Golodirsen, Skipping Exon 53

Sarepta Therapeutics completes submission of New Drug Application seeking approval of golodirsen in patients with DMD amenable to skipping exon 53 Press Release here Golodirsen has been studied for the treatment of exon 53 amenable patients, approximately eight percent of patients with DMD. Submission represents ongoing advancement of the company’s proprietary PMO RNA-based platform. Sarepta […]

FEELING BAD ABOUT THE HOLIDAYS SHOPPING FRENZY?

Giving Tuesday is your opportunity to give back. This is a day dedicated to generosity just as Black Friday and Cyber Monday are dedicated to shopping. Contributing to our cause will help someone afflicted Duchenne muscular dystrophy (DMD). It’s the moment in Canada and the whole world to celebrate giving back and supporting causes that matter to you. […]

POLARIS DMD, a phase 3 trial on edasalonexent

One of the objectives of our team is to inform you about new treatments. Here’s the latest news about Polaris DMD, the clinical trial of edasalonexent, developed by Catabasis. Thank you for sharing these articles in the DMD community.   Maria Mancini, vice president of clinical operations at Catabasis: We named the trial ‘Polaris’ because it […]

Tarantula venom for dmd

Here’s the latest news from the University of Buffalo (UB) about an experimental therapy called GsMTx4. Based on a molecule found in tarantula venom, this “protein” can prevent muscle mass loss and muscle damage in an animal model of Duchenne Muscular Dystrophy (DMD). Enjoy your reading and thank you for sharing these articles in the […]

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Patient registry demonstrating Translarna™ slows DMD progression

Here’s the latest news from PTC Therapeutics about Translarna™ (ataluren). In a recent press release, PTC announces that Translarna™ (ataluren) slows disease progression in children with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation. Enjoy reading and thank you for sharing these articles within the DMD community.   Analysis of data to date shows […]

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Positive results in the gene therapy micro-dystrophin trial to treat DMD

One of the objectives of our team is to inform you about new treatments. Here’s the latest news from Sarepta Therapeutics. They announce that at the 23rd International Congress of the World Muscle Society, Jerry Mendell, M.D., presented positive updated results from the four children dosed in the gene therapy micro-dystrophin trial to treat patients with […]

“Chimeric cells” for DMD?

Cells Restoring dystrophin in DMD We’ve set a goal for ourselves of informing you about new treatments for Duchenne muscular dystrophy (DMD). Here are some very promising results: new research conducted by Dr. Maria Siemionow, professor of orthopedic surgery in the UIC College of Medicine. Good reading and thank you for sharing these articles within the […]

Jérôme Frenette: OPG for DMD

Jérôme Frenette: OPG for DMD Professor Jérôme Frenette, himself lost two of his children due to a genetic disease. It’s, therefore, a decision from the heart that leads him to conduct today his research program on inherited diseases, like Duchenne Muscular Dystrophy (DMD). Professor Frenette understands very well the suffering of the people afflicted with […]

Understanding CRISPR/Cas9 with Jacques P. Tremblay

Canadian Research series This video series of interviews features Canadian scientists who work on solutions for Duchenne muscular dystrophy (DMD). We wish to connect you – the DMD community – with these scientists. You’ll be delighted to learn about their motivation and dedication to this cause. In turn, they appreciate knowing more about our community, […]