PPMD is thrilled to learn of the collaboration of Catabasis and Sarepta Therapeutics, two partners deeply committed to this community. This joint research collaboration will explore the potential combination therapy approach of NF-kB inhibition…Continue
Added by PPMD on September 29, 2016 at 9:21am — No Comments
Sarepta Therapeutics today announced the first patient dosed in the phase III clinical trial of SRP-4045 and SRP-4053 in patients with Duchenne muscular dystrophy amenable to exon 45 or 53 skipping.
Read the announcement from Sarepta:…Continue
Added by PPMD on September 28, 2016 at 6:30pm — No Comments
Santhera Pharmaceuticals today announced that the first patient has been enrolled in Santhera's phase III (SIDEROS) trial. The Phase III SIDEROS trial is designed to confirm the efficacy of Raxone in patients…Continue
Added by PPMD on September 28, 2016 at 8:54am — No Comments
Summit Therapeutics plc today announced it has received Fast Track designation from the US Food and Drug Administration (FDA) for ezutromid in the treatment of…Continue
Hillsdale College announced the creation of a merit-based scholarship to honor the late Ryan Newbanks, a young man who became close to the football program while battling Duchenne muscular dystrophy. The scholarship announcement coincides with the one-year anniversary of Newbanks’ death in 2015, and also with…Continue
Added by PPMD on September 20, 2016 at 9:00am — No Comments
Today, my sons are dancing! Today we ALL celebrate the first FDA approval in Duchenne. Today the FDA approved Exondys 51 (aka eteplirsen) injection, specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable…Continue
September is National Newborn Screening Awareness month and PPMD has been helping to put Duchenne on the newborn screening map — the WORLD map.
Our community understands the inherent benefits of early diagnosis. We know that knowledge…Continue
Added by Annie Kennedy on September 14, 2016 at 11:30am — No Comments
With the promise of drug approvals on the horizon, access to these therapies will be critical to our Duchenne community. PPMD has been leading work related to Duchenne and rare disease community access issues on multiple fronts.
The Institute for Clinical and…Continue
Added by Annie Kennedy on September 14, 2016 at 11:25am — No Comments
PPMD is excited to see the continued progress of vamorolone (VBP-15), with enrollment in the VISION-DMD Phase 2a Study now beginning. PPMD is proud to be an early funder of vamorolone and are hopeful that this compound will one day prove to be an alternative option for steroids.
Read the Press…Continue
Added by PPMD on September 8, 2016 at 1:35pm — No Comments
Added by Abby Bronson on September 8, 2016 at 9:43am — No Comments
The global Duchenne community will come together on September 7 for World Duchenne Awareness Day. This year a theme was chosen that is not only critical to changing the diagnostic odyssey for people with…Continue
Added by Annie Kennedy on September 1, 2016 at 1:30pm — No Comments
The global Duchenne community will come together on September 7 for World Duchenne Awareness Day. This year a theme was chosen that is not only critical to changing the diagnostic odyssey for people…Continue
Added by Ann Lucas on September 1, 2016 at 1:30pm — No Comments
The global Duchenne community will come together on September 7 for World Duchenne Awareness Day. This year a theme was chosen that is not only critical to changing the diagnostic odyssey for people with Duchenne…it is a theme…
Added by Kathi Kinnett on September 1, 2016 at 12:30pm — No Comments