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All Blog Posts Tagged 'Staff' (428)


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 — No Comments


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


Staff
What Does Independence Mean to the Duchenne Community?

July marks the 25th Anniversary of Americans with Disabilities Act (ADA), 50th Anniversary of Medicaid, and the dawning of a new age therapy development for our Duchenne community. But - as we all know - with new opportunities, come new challenges. As people with Duchenne grow up and grow into…

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Added by PPMD on July 4, 2015 at 12:31pm — 1 Comment


Staff
Revised Duchenne Anesthesia Recommendations 2015

Historically, people with Duchenne have been told that using inhaled anesthetics can cause malignant hyperthermia (“MH”; extremely high body temperature), a condition causing rhabdomyolysis (breakdown of skeletal muscle tissue causing the release of…

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Added by Kathi Kinnett on July 2, 2015 at 10:00am — No Comments


Staff
FDA Accepts BioMarin's NDA for Drisapersen; Sarepta Completes NDA for Eteplirsen

Big news in exon skipping therapy today! BioMarin announced this morning that the FDA has accepted their New Drug Application (NDA) for drisapersen and Sarepta announced that they have completed their NDA submission for eteplirsen.



We are such a fortunate…

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Added by PPMD on June 29, 2015 at 10:34am — No Comments


Staff
Webinar Recording: PF-06252616 (Anti-Myostatin)

On June 3, 2015, Dr. Michael Binks, Vice President for Rare Disease Clinical Research at Pfizer, presented information about the upcoming clinical trial of  PF-06252616. The webinar provides an overview of Pfizer’s approach to advancing the science of an anti-myostatin monoclonal antibody as an investigational compound for potential therapeutic use in muscle wasting diseases, such as Duchenne muscular dystrophy. Dr. Binks gives a presentation and answers submitted…

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Added by PPMD on June 25, 2015 at 3:30pm — No Comments


Staff
Urge your Senators to Cosponsor PFIA (S. 1597) from Home!

PPMD has been honored to lead the Duchenne community in developing patient-focused tools like our draft guidance and our benefit/risk surveys to help accelerate the FDA review of Duchenne treatments. Just last week, FDA issued its landmark draft guidance on Duchene therapy development, a document heavily…

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Added by Ryan Fischer on June 19, 2015 at 1:00pm — No Comments


Staff
Update on 21st Century Cures Initiative



Over the last year, we have been telling you about the 21st Century Cures Initiative, a comprehensive look launched by Congress to explore what could be done to accelerate the…

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Added by Annie Kennedy on June 15, 2015 at 2:07pm — No Comments


Staff
Accessing Drugs Once They’re Approved

As the approval of therapies in Duchenne becomes more of a reality for our community, access becomes critical. Potential challenges with insurance coverage and approvals, as well as specialty pharmacy access begin to become a very real concern. And typically new and innovative therapies marketed to…

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Added by Annie Kennedy on June 15, 2015 at 1:30pm — No Comments


Staff
PPMD Listens

PPMD Listens. We have listened to patients, to families, to industry, and to providers. We have listened, and we have acted quickly and effectively, to help to improve your care.

  • In 2012, after listening to what care and services patients and families were not receiving, we convened the…
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Added by Kathi Kinnett on June 15, 2015 at 12:30pm — No Comments


Staff
Parent Project Muscular Dystrophy Names Duke Medicine Certified Duchenne Care Center

Parent Project Muscular Dystrophy is excited to name Duke Children’s Hospital & Health Center (Duke Children’s) a…

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Added by PPMD on June 12, 2015 at 9:29am — No Comments


Staff
PPMD's 2015 Connect Conference: Thank You to Our Sponsors!

Today, we are blessed with an overwhelming number of industry and corporate partners who have committed themselves to the fight to end Duchenne. This kind of commitment was unheard of when PPMD started 21 years ago. Because of the dedication of these companies and others, we are stronger as a community. We are closer to ending Duchenne.…

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Added by PPMD on June 11, 2015 at 10:30am — No Comments


Staff
FDA Releases Draft Guidance for Duchenne

Amazing things happen when this community works together, when our messages are consistent, and our goals are in sync. We work together, we change the landscape, we make history.

 

Today the FDA has…

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Added by Pat Furlong on June 9, 2015 at 9:00am — 2 Comments

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