February 25, 2019 Santhera Pharmaceuticals announces results from the SYROS study. The primary objective of this study was to evaluate the long-term evolution of the respiratory function in patients who maintained treatment with idebenone for up to 6 years compared to their preceding off-idebenone period. Respiratory Function in DMD In boys and men […]
The success of CRISPR in a model of Duchenne muscular dystrophy Original story from Duke University Researchers at Duke University have shown that by using genome editing technology, CRISPR can safely and stably correct a genetic condition such as Duchenne Muscular Dystrophy (DMD). The study appears in the journal Nature Medicine. Links here CRISPR/Cas9 […]
PTC124-GD-041-DMD (Also known as study 041) The clinical trial of ataluren in nonsense mutation Duchenne muscular dystrophy (NMDMD) Study 041 is a new clinical trial for boys and young men who are living with nonsense mutation Duchenne Muscular Dystrophy (nmDMD). Who Can Enter Study 041? To ensure that participants who enroll in Study 041 get […]
Sarepta Announces FDA Acceptance of Golodirsen (SRP-4053) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53 Press release here: Sarepta.com Sarepta Therapeutics, Inc. announced the Food and Drug Administration, Division of Neurology had accepted its New Drug Application (NDA) seeking accelerated approval for golodirsen (SRP-4053) and provided a regulatory […]
The zebra is used as a symbol for rare diseases since about 1940. This comes from a quote by Dr. Theodore Woodward: “When you hear hoofbeats, think horses, not zebras.” and “When you hear hoofbeats behind you, don’t expect to see a zebra.” This is the metaphor Dr. Woodward used to teach students basic concepts about […]
Because it manifests itself only rarely, Duchenne muscular dystrophy (DMD) falls within the rare diseases category. It is also a treatment orphan disease. Since this is February, it is pertinent for us to tell you about it. Rare Disease Day takes place on the last day of February each year. The primary objective of Rare Disease Day is to raise awareness among […]
Victor was diagnosed with Duchenne DMD is an incurable and 100% fatal disease. Duchenne muscular dystrophy is a rare genetic disorder that affects mainly boys. The progression of DMD is unforgiving: the body’s muscles gradually weaken, leaving the boys in a wheelchair at the age of 12, with a life expectancy of 20 to 30 years. Today there […]
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 […]
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 […]
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. […]
