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All Blog Posts Tagged 'Staff' (439)


Staff
BioMarin: Quarterly Update to the Duchenne Community (August 2015)

In an effort to provide regular communication on BioMarin's Duchenne muscular dystrophy programs to the Duchenne community, the company is circulating a quarterly update. This update is the first of its kind for BioMarin, and they welcome your feedback to this new communication channel, which you may do by…

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Added by PPMD on August 17, 2015 at 12:30pm — No Comments


Staff
Summit Therapeutics Announces Phase 1B Modified Diet Clinical Trial Achieves Primary Objective In Duchenne Muscular Dystrophy

Summit Therapeutics plc announced that its Phase 1b modified diet clinical trial of SMT C1100 for the treatment of Duchenne met its primary objective with half of the patients who received the higher dose of SMT C1100 achieving desired plasma levels while following specific dietary guidance.  Based on these results, Summit will advance SMT C1100 into a Phase 2 open-label clinical trial. Congratulations to our friends at Summit and we look forward to hearing more about next…

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Added by PPMD on August 17, 2015 at 10:25am — 1 Comment


Staff
Marathon Pharmaceuticals to Begin NDA Process for Deflazacort as a Potential Treatment for Duchenne

Last week, Marathon Pharmaceuticals announced that the company will begin the New Drug Application (NDA) process for deflazacort as a potential treatment for patients…

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Added by PPMD on August 12, 2015 at 4:07pm — No Comments


Staff
PPMD’s DuchenneConnect to Receive $1.4 Million Funding Award from PCORI

(pictured: DuchenneConnect's Family Advisors at PPMD's 2015 Connect Conference)



DuchenneConnect
, led by PPMD, has been approved for…

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Added by Ann Lucas on August 12, 2015 at 12:04pm — No Comments


Staff
What is an Ad Comm?

Ad Comm. It’s a term our community will be hearing a lot this fall as companies get closer to the possible approval of treatments for Duchenne. But what is an Ad Comm and how does it involve and affect you or your…

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Added by Ryan Fischer on August 11, 2015 at 12:00pm — No Comments


Staff
Imperatives for DUCHENNE MD: A simplified guide to care for Duchenne published in PLOS

Providers care for many people, with many diagnoses – far too many for any one health care provider to be an expert in them all. Any busy provider would find it difficult to have the time to look over a two-volume document like the Care Considerations [PDF], or even a several-page document like the…

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Added by Kathi Kinnett on August 11, 2015 at 9:30am — No Comments


Staff
One month away from World Duchenne Awareness Day!

People around the world will help participate in the second annual World Duchenne Awareness Day on September 7th. There are a variety of ways you can get involved with this international effort – how will you…

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Added by Ryan Fischer on August 7, 2015 at 10:30am — No Comments


Staff
PPMD Submits Comments on FDA's Draft Guidance on Duchenne

We are coming to the end of the 60 day open comment period for the FDA’s draft guidance on Duchenne. PPMD submitted our comments to the agency yesterday –…

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Added by PPMD on August 6, 2015 at 11:09am — No Comments


Staff
Action ALERT: Urge your Senators to Cosponsor S. 1597, the Patient-Focused Impact Assessment Act!

Call and email your Senators today! The Food and Drug Administration’s efforts to support meaningful patient engagement, also known as Patient-Focused Drug Development (PFDD), includes the strengthening of existing tools and creation of new ones needed to fill gaps, policies to allow for greater engagement of external experts in the review process, and a…

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Added by PPMD on August 5, 2015 at 12:57pm — No Comments


Staff
Different Together: PPMD Announces the Duchenne Regulatory Science Consortium

Signal to Noise

This is a measure used in science and engineering to compare the level of a desired signal (a measured outcome) to the background noise. In a way, this is the question of what information carries weight/is meaningful and what information clouds the issue.



While this sounds really complicated, we have witnessed…

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Added by John D. Porter, PhD on August 3, 2015 at 9:00am — No Comments


Staff
Changes to Deflazacort Ordering Process (via Masters Pharmaceuticals)

Masters now has an increasing supply of Deflazacort (a steroid available to treat Duchenne outside the US).…

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Added by PPMD on July 27, 2015 at 4:00pm — 1 Comment


Staff
PPMD's 2015 Connect Conference Resources & Recordings - Part 3 (Saturday)

Presentations from Day 3 of PPMD’s 21st Annual Connect Conference in Washington, D.C. are now available for download. If you were unable to attend this year’s conference or if you want to replay a presentation that you found particularly useful, we have been sharing all of the presentations online over the…

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Added by PPMD on July 21, 2015 at 12:31am — No Comments


Staff
Participate in ABLE National Resource Center Survey

Since the enactment of the Stephen Beck, Jr., Achieving a Better Life Experience Act of 2014 (ABLE Act) in December 2014, PPMD has been working with leading…

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Added by Annie Kennedy on July 20, 2015 at 12:00pm — No Comments


Staff
PPMD Awards $70,000 Grant to Talem Technologies as Part of Robotics Initiative

Grant Will Be Used to Support Pilot Study for X-Ar Exoskeletal Arm Technology



Parent Project Muscular Dystrophy has awarded Talem Technologies a grant for over $70,000 to support a pilot study of their…

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Added by PPMD on July 20, 2015 at 10:00am — No Comments


Staff
PPMD's 2015 Connect Conference Resources & Recordings - Part 2 (Friday)

Presentations from Day 2 of PPMD’s 21st Annual Connect Conference in Washington, D.C. are now available for download. If you were unable to attend this year’s conference or if you want to replay a presentation that you found particularly useful, we will be sharing all of the presentations over the next…

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Added by PPMD on July 17, 2015 at 11:30am — No Comments


Staff
PPMD's 2015 Connect Conference Resources & Recordings - Part 1 (Thursday)

The first batch of presentations from PPMD’s 21st Annual Connect Conference in Washington, D.C. are available for download. If you were unable to attend this year’s conference or if you want to replay a presentation that you found particularly useful, we will be sharing all of the presentations over…

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Added by PPMD on July 14, 2015 at 9:00am — No Comments


Staff
PPMD's 2015 Duchenne/Becker Transitions Meeting Recap

“We are the population that was hoped for — and now we’re here.”

- L. Vance Taylor



This statement made by a close friend of mine perfectly captures the spirit that satiated the room at the PPMD Duchenne/Becker Transitions Meeting which took place at…

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Added by Annie Kennedy on July 13, 2015 at 11:00am — 1 Comment


Staff
Download PPMD's 2015 Connect Conference Booklet

PPMD’s 2015 Connect Conference booklet contained over 40 pages of information on what’s currently going on in Duchenne research & clinical trials. We encourage you to browse the digital version of the booklet if you were unable to attend. And stay tuned for more research updates as we'll be posting recordings of presentations from the conference over the next few…

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Added by PPMD on July 11, 2015 at 12:00pm — No Comments


Staff
Action ALERT: Urge Congress to Vote YES on 21st Century Cures Act

For over a year, PPMD has been working with Congress on landmark legislation called 21st Century Cures. The bill aims to speed the discovery, development, and delivery of therapies for diseases that have an unmet medical need like Duchenne. We have been thankful to…

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Added by PPMD on July 8, 2015 at 11:17am — 1 Comment


Staff
Catabasis Pharmaceuticals Receives FDA Fast Track Designation for CAT-1004 for the Treatment of Duchenne Muscular Dystrophy

PPMD is excited that Catabasis has received Fast Track designation for CAT-1004 and we are proud to be an early supporter of this promising therapy.

Catabasis Pharmaceuticals Receives FDA Fast Track Designation for CAT-1004 for the Treatment of Duchenne Muscular Dystrophy



CAMBRIDGE, MA, July 6, 2015…

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Added by PPMD on July 7, 2015 at 1:30pm — 1 Comment

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