Microdystrophin gene transfer trial on Hold

Solid Biosciences Announces Clinical Hold On SGT-001 microdystrophin gene transfer Clinical Phase I/II Clinical Trial for Duchenne Muscular Dystrophy.  Solid Biosciences Inc. announced it had received notification from the U.S. Food and Drug Administration (FDA) that IGNITE DMD, its Phase I/II clinical trial for microdystrophin gene transfer in Duchenne muscular dystrophy (DMD), has been placed on Clinical […]


Canadian Research osteoprotegerin (OPG)

Meeting with professor Jérôme Frenette about osteoprotegerin (OPG). We are producing a video series of interviews with Canadian researchers working on DMD. We have met a number of professors and our videos will be coming up soon.  We wish to connect you, the DMD community, with the professors. You will be delighted to know their deepest […]

Cycling across Canada to cure DMD

What’s it like to live with DMD? As a parent, to face the reality that your child has a fatal disease? As a person with DMD, to face an inevitable, premature death in your twenties? As a sibling, relative or friend, to face mourning your loss? The journey is beyond words. Our Web short documentary […]

Decision from the FDA’s Office for Ataluren

Today, PTC Therapeutics, Inc, announced that the Office of New Drugs of the U.S. Food and Drug Administration has reiterated the FDA’s prior position and denied PTC’s appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren. In its letter, the Office of New Drugs recommended a possible path […]

Short Docs series: Portrait of Duchenne Canada

Our web short documentary series, “Portrait of Duchenne Canada”, will be coming son!  We will present the experiences of families and children with DMD across Canada.What’s it like to live with Duchenne muscular dystrophy (DMD)? As a parent, to face the reality that your child has a fatal disease? As a person with DMD, to face […]

February is the Rare Disease Month

February is 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.    A few distinctions … A rare disease is defined as a disease that affects less […]