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October 2016 Blog Posts (23)


Staff
Deflazacort Access: Navigating Potential Changes to Your Prescription

In an effort to answer some of the questions that have arisen with the potential FDA approval of deflazacort in the U.S., PPMD has worked with both Masters and Marathon to put together the resource below, which we hope will provide clarity.…

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Added by Kathi Kinnett on October 31, 2016 at 2:00pm — No Comments


Staff
The Assistance Fund Launches Duchenne Program to Support Access to Exondys 51

Patient Assistance programs are typically non-profit organizations sponsored by drug companies, patient advocacy organizations, and civic groups that establish disease specific programs to facilitate access to brand-name medications.



In the case of…

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Added by Annie Kennedy on October 28, 2016 at 11:00am — No Comments


Staff
PPMD Designates Center for Duchenne Muscular Dystrophy at UCLA, a Certified Duchenne Care Center

PPMD Awards Twelfth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care

 

The Center for Duchenne Muscular Dystrophy at the…

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Added by PPMD on October 26, 2016 at 3:02pm — No Comments


Staff
PPMD Designates Center for Duchenne Muscular Dystrophy at UCLA, a Certified Duchenne Care Center

PPMD Awards Twelfth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care

 

The Center for Duchenne Muscular Dystrophy at the…

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Added by PPMD on October 26, 2016 at 3:02pm — No Comments


Staff
The Latest Update on Translarna from PTC Therapeutics

The journey has been long. From PTC Therapeutics' first presentation about targeting a ‘stop sign’ within the dystrophin mutation to PTC 124 to ataluren and now, Translarna. The journey has been a long one.

The team at PTC are pioneers, the company…

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Added by Pat Furlong on October 26, 2016 at 12:30pm — 1 Comment


Staff
Solid Biosciences Granted U.S. and E.U. Orphan Drug Designations for Lead Gene Therapy Candidate for Duchenne Muscular Dystrophy

Solid Biosciences and its subsidiary, Solid GT, announced today that the FDA and the European Commission have granted Orphan Drug designations for the company’s gene therapy candidate, SGT-001,…

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Added by PPMD on October 24, 2016 at 9:00am — No Comments


Staff
Register for PPMD's 2017 Advocacy Conference | February 12-14 in Washington, DC

 

You are invited to join us February 12-14, 2017 in Washington D.C. for what is going to be another critical year in Duchenne advocacy.

 

Register Now

 

2016 marked a tremendous…

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Added by Ryan Fischer on October 20, 2016 at 1:00pm — No Comments


Staff
[FOR CLINICIANS] The Emergent & Acute Management of Patients Living with Duchenne Muscular Dystrophy

This presentation, "The Emergent and Acute Management of Patients Living with Duchenne Muscular Dystrophy," was produced in an effort to educate clinicians in the care of individuals and families living with Duchenne.



The…

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Added by Kathi Kinnett on October 19, 2016 at 3:30pm — No Comments


Staff
Duchenne Research Update: October 2016

When I first joined the Duchenne community in 2009 at Children’s National Medical Center, I remember thinking that I was walking into a critical juncture in Duchenne history. There were three compounds in the clinic (drisapersen, ataluren, and eteplirsen aka Exondys…

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Added by Abby Bronson on October 18, 2016 at 1:00pm — No Comments


Staff
DuchenneConnect Twitter Chat on Thursday, October 20 at 1PM EST

Join us for our first DuchenneConnect Twitter chat on Thursday, Oct 20, 2016 from 1-2:00 PM EST. We will be using the hashtag #DuchenneConnectTC.



Our first chat will cover the…

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Added by Ann Lucas on October 17, 2016 at 11:30am — No Comments


Staff
PTC Therapeutics Provides Regulatory Update on Translarna™

PPMD is disappointed to learn that the FDA denied PTC Therapeutics' first appeal of the refuse to file letter issued in February. As we understand it, this is a process and may involve additional appeals. PPMD will be…

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Added by PPMD on October 17, 2016 at 10:24am — No Comments


Staff
PPMD Demands Anthem Reconsider Coverage of Approved Drug

It is hard to imagine anything worse than receiving the diagnosis of Duchenne. The dates and odysseys that brought us into this community differ, but the fervor we share to slow and stop the progression of this disease as it marches through our children is unparalleled in any…

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Added by Pat Furlong on October 14, 2016 at 12:02pm — No Comments


Staff
Connect with PPMD in 2017!

Can you believe 2017 is just around the corner? It will be here before you know it and PPMD is busy putting together an exciting list of events that will continue to connect and inform our Duchenne community. It’s already a packed new year!

 

Check out where your PPMD family plans to be in 2017 and mark your calendars!…

Upcoming Events

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Added by PPMD on October 14, 2016 at 9:30am — No Comments


Staff
ABLE Accounts: Improvements Moving Through Congress & Determining Which State’s Program is Best for You?

With the initiation of 4 states ABLE programs already begun and Oregon having just anounced that their program will launch in December, any qualified individual (meaning most families with Duchenne) can open their accounts in 2016, if interested. We have previously shared information…

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Added by Annie Kennedy on October 13, 2016 at 8:37pm — No Comments


Staff
PPMD Awards $239,000 Research Grant to Dr. Terence Partridge of Children’s National Medical Center

Parent Project Muscular Dystrophy (PPMD) has awarded a $239,000 grant to Dr. Terence Partridge, Professor of Systematic Integrative Biology, Research Center for Genetic Medicine at Children's National Medical Center. This grant will help Dr. Partridge and his team continue their work in…

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Added by PPMD on October 13, 2016 at 11:31am — No Comments


Staff
Calling All Adults with Duchenne/Becker – Join the 2017 PPMD Adult Advisory Committee!

Ever wished you could change the world – or at least your piece of it? Been looking for a way to engage a bit more? Have personal experience that you’d like to see be better reflected in national policy and program efforts?



PPMD is seeking adult members of our Duchenne/Becker…

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Added by Annie Kennedy on October 12, 2016 at 11:30am — No Comments


Staff
[Webinar Takeaways] Preparing for Access to Approved Therapies

Last week, PPMD began a webinar series on access – an exciting new chapter for our Duchenne community. But we are just at the beginning of this chapter and there is lots to explore.



We knew this day was coming –…

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Added by Annie Kennedy on October 11, 2016 at 3:00pm — No Comments


Staff
Anthem and Exondys 51 (Eteplirsen)

PPMD is deeply troubled to learn that health insurer Anthem will not be covering Exondys 51, stating the following position:

“The use of eteplirsen is considered investigational and not medically necessary for all indications, including but not limited to the treatment of Duchenne muscular dystrophy.”

This is not an…

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Added by PPMD on October 7, 2016 at 2:30pm — No Comments


Staff
PTC Therapeutics Announces New Analyses that Support the Potential Benefit of Ataluren in Preserving Lung Function in Non-Ambulatory Nonsense Mutation Duchenne Muscular Dystrophy Patients

PTC Therapeutics, Inc. today announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory people with nonsense mutation Duchenne. PPMD continues to urge the FDA to provide PTC with a full review and advisory committee meeting.…

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Added by PPMD on October 6, 2016 at 9:15am — No Comments


Staff
PPMD's DuchenneConnect Team Presents at Annual Genetic Conference



Last week, Ann Lucas and I had the honor of presenting an Educational Breakout Session focused on Duchenne at the National Society of Genetic Counselors (NSGC) Annual Education Conference in Seattle. Lauren Morgenroth from TRiNDS also presented during the…

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Added by Ann Martin on October 5, 2016 at 1:00pm — No Comments

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