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September 2016 Blog Posts (13)


Staff
Catabasis Pharmaceuticals & Sarepta Therapeutics Announce a Joint Research Collaboration in Duchenne

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…

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Added by PPMD on September 29, 2016 at 9:21am — No Comments


Staff
Sarepta Therapeutics Announces First Patient Dosed in Phase III Clinical Trial of SRP-4045 & SRP-4053

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:…

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Added by PPMD on September 28, 2016 at 6:30pm — No Comments


Staff
Santhera Starts Phase III Study (SIDEROS) with Raxone in Patients with Duchenne Using Glucocorticoids

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…

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Added by PPMD on September 28, 2016 at 8:54am — No Comments


Staff
Summit Receives Fast Track Designation from FDA for Ezutromid in the Treatment of Duchenne

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…

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Added by PPMD on September 26, 2016 at 9:30am — 1 Comment


Staff
Hillsdale College Announces Scholarship to Honor Ryan Newbanks

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…

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Added by PPMD on September 20, 2016 at 9:00am — No Comments


Staff
FDA Grants Accelerated Approval to First Drug for Duchenne Muscular Dystrophy

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…

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Added by Pat Furlong on September 19, 2016 at 1:30pm — 4 Comments


Staff
September is National Newborn Screening Awareness Month



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…

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Added by Annie Kennedy on September 14, 2016 at 11:30am — No Comments


Staff
PPMD Continues to Lead Work on Critical Duchenne Access Issues

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. …

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Added by Annie Kennedy on September 14, 2016 at 11:25am — No Comments


Staff
ReveraGen BioPharma Inc initiates VISION-DMD Phase 2a Study for Treatment of Duchenne

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…

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Added by PPMD on September 8, 2016 at 1:35pm — No Comments


Staff
PPMD Awards $200,000 Grant to Joshua Selsby, PhD of Iowa State University

Quercetin /ˈkwɜːrstn/ is a flavonol found in many fruits, vegetables, leaves and grains. It can be used as an ingredient in supplements, beverages, or foods. Source:…

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Added by Abby Bronson on September 8, 2016 at 9:43am — No Comments


Staff
The Importance of Early Diagnosis: Eliminating the Diagnostic Odyssey

The global Duchenne community will come together on September 7 for World Duchenne Awareness Day. This year a theme was chosen that is not…

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Added by Annie Kennedy on September 1, 2016 at 1:30pm — No Comments


Staff
The Importance of Early Diagnosis: Improving the Lives of Carries & Their Families

The global Duchenne community will come together on September 7 for World Duchenne Awareness Day. This year a theme was chosen that is not…

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Added by Ann Lucas on September 1, 2016 at 1:30pm — No Comments


Staff
The Importance of Early Diagnosis: Ensuring Quality Care



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…

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Added by Kathi Kinnett on September 1, 2016 at 12:30pm — No Comments

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