CRISPR & Duchenne muscular dystrophy
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 [...]
CLINICAL TRIAL OF ATALUREN IN CANADA
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 [...]
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 [...]
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 [...]
