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All Blog Posts Tagged 'Staff' (20)


Staff
BREAKING NEWS: 21st Century Cures Crossed Over the Finish Line!

The Duchenne Community’s Voice Was Essential Every Step of the Way & Contains PPMD’s Provision to Strengthen Patient Focused Drug Development Efforts



To everyone in our PPMD community who called, reached out, emailed, and met with your elected…

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Added by Annie Kennedy on December 7, 2016 at 2:30pm — No Comments


Staff
PPMD’s #EverySingleOne Tour: Durham

Duke University marked our final stop on PPMD’s 2016 Every Single One Tour, held on November 2. We were thrilled to be holding another tour stop at one of…

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Added by Ryan Fischer on December 7, 2016 at 11:04am — No Comments


Staff
Light up the Lines!! Urge Your Senators to Vote YES on 21st Century Cures!

We need you to call your two US Senators TODAY with one simple message – VOTE YES ON 21st CENTURY CURES!!

 

With just days remaining in the current Congress, this bill represents the extraordinary efforts of our PPMD community’s work and outreach with…

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Added by Ryan Fischer on December 5, 2016 at 10:07am — No Comments


Staff
Your gift to CRISPR will be Matched!

We have so much momentum. Let’s seize this moment.





First of all, let me say THANK YOU! Your gift to PPMD on #GivingTuesday helped us surpass our goal, and we could not be more grateful. It is a wonderful way to kick off our holiday season—and we wanted to make sure you were among the…

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Added by Pat Furlong on December 1, 2016 at 9:30am — 1 Comment


Staff
ACTION ALERT: Urge Congress to Vote ‘YES’ on 21st Century Cures Act This Week!

After two years of unwavering leadership from Congressional Champions dedicated to Duchenne and other rare diseases — a final version of the landmark 21st Century Cures Act was released over Thanksgiving weekend.

With just days remaining in the current Congress, this bill represents the extraordinary…

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Added by Ryan Fischer on November 28, 2016 at 12:30pm — No Comments


Staff
A truly thankful moment

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Added by Pat Furlong on November 23, 2016 at 5:30pm — No Comments


Staff
Summit Enrolls U.S. Patients into Phaseout DMD, a Phase 2 Clinical Trial of Ezutromid in Duchenne

Summit Therapeutics plc announced today that it has enrolled its first patients at trial sites in the US into PhaseOut DMD, a Phase 2 clinical trial of ezutromid in Duchenne. The 48-week, open-label (meaning all participants receive ezutromid) Phase 2 trial is enrolling up to 40 boys total between their 5th…

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Added by PPMD on November 16, 2016 at 9:00am — No Comments


Staff
Duchenne Carrier Study at Nationwide Children’s Hospital

Frequently, I get calls from mothers/sisters/aunts of people living with Duchenne about issues related to being a carrier of Duchenne:



  • Should they have carrier…
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Added by Kathi Kinnett on November 14, 2016 at 11:00am — 2 Comments


Staff
CHMP Recommends Renewal of Translarna's™ Marketing Authorization Based on Continued Positive Benefit-Risk Assessment

PTC Therapeutics, Inc. today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna(TM) (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients…

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Added by PPMD on November 11, 2016 at 9:30am — No Comments


Staff
2016 Flu Vaccination Recommendations

Everyone, six months of age and older, needs to be vaccinated against influenza (flu), and protecting people living with with Duchenne is especially important. A …

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Added by Kathi Kinnett on November 9, 2016 at 2:30pm — 2 Comments


Staff
Akashi Provides HT-100 Clinical Trial Update

In February, dosing and new patient enrollment in all cohorts of the HALO trial, a study evaluating the compound HT-100 in patients with Duchenne, was suspended after one of the patients in the trial, receiving 60μg/kg/d (the highest dose in the study), passed away after experiencing serious, life-threatening health issues. Since then, the company has been working with the…

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Added by PPMD on November 7, 2016 at 11:30am — No Comments


Staff
Pfizer Shares Update on Phase II Clinical Trial of Domagrozumab (PF-06252616) in Duchenne

Pfizer has provided the following update regarding the on-going Phase II clinical trial of domagrozumab (PF-06252616), a myostatin inhibitor, in people with Duchenne.



Notably, the age range for this clinical study has been expanded from 6 to <10 years of age to 6 to <16 years of age. Additional sites have been added in the US, United Kingdom, Poland, Bulgaria, and Australia.



Pfizer also reports that enrollment of the phase 2 study of…

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


Staff
Understanding Insurance Determinations



With the accelerated approval of EXONDYS 51™ (aka eteplirsen) by the FDA in September, the Duchenne community has been quickly…

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Added by Annie Kennedy on November 2, 2016 at 12:30pm — No Comments


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

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