Deflazacort over prednisone/prednisolone?
Here’s the latest news from PTC Therapeutics about Deflazacort (EMFLAZA®). A publication of data in Muscle & Nerve comparing the efficacy and safety of deflazacort and prednisone/prednisolone from the placebo arm of the ACT DMD study has been published in Muscle & Nerve. Enjoy reading and thank you for sharing these articles within the DMD community. […]
A call to resilience
A text written by Guylaine Rivard This is a text that has been originally published in the group Les mamans Duchenne. On June 18th in 2009, it’s been almost ten years ago today that the DMD diagnosis for Félix fell on us. I say dropped on us, but it was more or so as if I […]
Negative opinion for EXONDYS® in Europe
Here’s the latest news from Sarepta Therapeutics press release about EXONDYS® (eteplirsen). EXONDYS® (eteplirsen) is designed to treat patients with Duchenne muscular dystrophy (DMD) amenable to skipping exon 51 of the dystrophin gene. Enjoy reading and thank you for sharing these articles within the DMD community. Sarepta receives negative CHMP opinion for EXONDYS® […]
Solid Biosciences announces new preclinical data about gene transfer
One of the objectives of our team is to inform you about new treatments. Here’s the latest news from a recent Solid Bioscience press release about SGT-001, the Company’s lead microdystrophin gene transfer candidate. Enjoy reading and thank you for sharing these articles within the DMD community. Solid Biosciences announces new preclinical data […]
Positive news for Translarna™ (ataluren)
One of the objectives of our team is to inform you about new treatments. Here’s the latest news from PTC Therapeutics press release about Translarna ™ (ataluren). Enjoy reading and thank you for sharing these articles within the DMD community. CHMP Adopts Positive Opinion for the Expansion of the Translarna ™ (ataluren) Label to […]
