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New drug for DMD being reviewed by the FDA

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-striped ribbon as a symbol for rare diseases

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 […]

February, the rare disease month

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 […]

And if art became a cure?

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 […]

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 […]