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All Blog Posts Tagged 'Staff' (667)


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


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

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