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All Blog Posts Tagged 'PPMD Blog' (754)


Staff
ACTION ALERT: Stop the Graham-Cassidy proposal in the Senate

The Senate is considering another effort to repeal the Affordable Care Act (ACA) before the end of the month. Last week, Senators Lindsey Graham (R-SC), Bill Cassidy (R-LA), Dean Heller (R-NV), and Ron Johnson (R-WI) released a new plan to repeal and replace the ACA. This proposal is commonly referred to as…

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Added by Annie Kennedy on September 19, 2017 at 10:00am — No Comments


Staff
2017/2018 Flu Vaccination Recommendations

Everyone, six months of age and older, needs to be vaccinated against influenza (flu) by the end of October, if possible. Protecting people living with Duchenne is especially important. A …

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Added by Kathi Kinnett on September 18, 2017 at 10:30am — No Comments


Staff
PPMD Designates Nemours/Alfred I duPont Hospital for Children a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named Nemours/Alfred I duPont Hospital for Children (Nemours) in Wilmington, Delaware our 17th Certified Duchenne Care Center.

PPMD’s Certified Duchenne Care Center Program,…

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Added by PPMD on September 14, 2017 at 8:30pm — No Comments


Staff
PPMD Lends Leadership to NCATS Rare Disease Research Toolkit Development

There are some days when I feel like our community is in its infancy. When I look at where we are in terms of access, therapeutic options, combinations, care implementation…



But more often than not, I am overwhelmed with gratitude for the infrastructure and resources…

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Added by Annie Kennedy on September 13, 2017 at 5:00pm — No Comments


Staff
PPMD’s #EverySingleOne Tour: Charleston, WV

We kicked off our fall Every Single One Tour stops in West Virginia this past weekend! We appreciate all of the families, speakers, and volunteers that spent the day with us in…

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Added by Kathi Kinnett on September 13, 2017 at 4:30pm — No Comments


Staff
MD Community Moves Forward ICD Code Nomination for Duchenne/Becker

Paperwork – oh the paperwork!

Each time you visit a physician’s office or hospital, we leave with piles of forms and papers detailing the visit. The providers seen, the prescriptions written, results from tests conducted, instructions for follow up, and more. And each of those pieces of…

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Added by Annie Kennedy on September 13, 2017 at 10:00am — No Comments


Staff
Reminder: Deadline to Submit Written Comments for Ataluren Ad Comm is Thursday, September 14

The deadline to submit a written comment for the FDA Advisory Committee Meeting on ataluren is this upcoming Thursday, September 14th.

 

To make a…

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Added by PPMD on September 12, 2017 at 9:30am — No Comments


Staff
The 2018 End Duchenne Tour – Coming to a City Near You!

PPMD is excited to announce next year’s lineup of areas we will visit as part of our End Duchenne Tour!



Combining each of the pillars that make up PPMD’s mission, the End Duchenne Tour brings updates on research, advocacy, and care to cities across the…

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Added by PPMD on September 8, 2017 at 5:36pm — No Comments


Staff
Sarepta Therapeutics Announces Positive Results from 4053-101 Study

Sarepta Therapeutics, Inc. today announced muscle biopsy results from its 4053-101 study, a Phase 1/2 first-in-human study conducted in Europe to assess the safety, tolerability, pharmacokinetics, and efficacy of golodirsen…
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Added by PPMD on September 6, 2017 at 10:00am — No Comments


Staff
Roche Provides Community Update on BMS-986089 (RG6206)

Earlier this year, Bristol-Myers Squibb (BMS) announced it had entered into an agreement to license BMS-986089 (RG6206), an anti-myostatin adnectin in development for Duchenne muscular dystrophy, to Roche. Today, the Roche team has…

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Added by PPMD on September 5, 2017 at 9:30am — No Comments


Staff
FDA Announces Approval of First Gene Therapy in the United States

Yesterday, Kymriah, a gene therapy made by Novartis that treats an aggressive type of leukemia was approved for children and young adults. The new commissioner of the FDA, Dr. Scott Gottlieb,…

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Added by Abby Bronson on August 31, 2017 at 1:00pm — No Comments


Staff
Care Complications – Going Beyond the More Common Elements of Care in Duchenne

Every year at PPMD's Annual Connect Conference, we try to put together the best topics, with the most informed speakers, to address elements of care at…

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Added by Kathi Kinnett on August 28, 2017 at 11:30am — No Comments


Staff
Webinar Q&A: Gene Therapy for Duchenne

With trials in gene therapy on the horizon, and terms like "cassette", "construct", "promoter", and "transduce" being thrown around, we want to ensure that you, our Duchenne Community, has all the knowledge you need to navigate the world of gene therapy.



Earlier this…

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Added by Abby Bronson on August 23, 2017 at 8:00pm — No Comments


Staff
How You Can Participate in the Ataluren Ad Comm

The Duchenne community is now preparing for the September 28 Advisory Committee Meeting for PTC’s therapy ataluren. Whether submitting written…

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Added by PPMD on August 22, 2017 at 11:00am — No Comments


Staff
PPMD's Bone Health Workshop Published

May 12-13, 2016, PPMD convened a workshop of 48 opinion leaders, research experts and clinicians from the US and Canada, on the subject of Duchenne and bone biology. Bone health has long been a…

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Added by Kathi Kinnett on August 14, 2017 at 10:00pm — No Comments


Staff
Understanding Clinical Trials in Duchenne

Hope is critical to survival. Hope helps us get through those times of darkness with the thought that there is something new on the horizon, a potential new destination. Hope enables us to focus on meeting challenges even in the midst of adversity. Hope allows us to see that there might be…

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Added by Ann Lucas on August 7, 2017 at 2:00pm — No Comments


Staff
PDUFA VI Passes the Senate – Now Moves to President’s Desk to be Signed Into Law

The PDUFA VI bill – known as FDA Reauthorization Act in the House (FDARA) – passed the House of Representatives early in July with strong bi-partisan support. It then moved to the Senate but consideration was delayed until after the Senate healthcare reform deliberations were completed. Today, though, we are excited that a companion bill (mirroring the House version) passed in the Senate! It will now move to the President’s desk where it is expected…

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Added by Annie Kennedy on August 3, 2017 at 8:30pm — No Comments


Staff
UPDATE - Action Alert: Your Calls Are Still Needed! Tell Your Senator to Vote NO on the Senate Health Bill

Yesterday the Senate voted 51-50 to move forward on healthcare reform legislation -- but no specific bill was actually voted on. The Senate has committed to passing something this week. Once passed, the Senate bill will then have to return to the House for passage or conferencing (if it differs from…

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Added by Annie Kennedy on July 26, 2017 at 9:00am — No Comments


Staff
Pulmonary Outcomes Workshop Summary

In April 2016, PPMD convened a workshop to assess the measures used to evaluate pulmonary strength, air flow and lung volumes. Thanks to the tireless effort of Dr. Jonathan Finder, this summary has now…

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Added by Kathi Kinnett on July 17, 2017 at 9:35am — No Comments

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