All Blog Posts (1,822)


Staff
PPMD to Host Landmark Duchenne Patient-Focused Compass Meeting

Join us in person or virtually!

As part of this year's Advocacy Conference (March 4-6, 2018), PPMD will host the Duchenne…

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Added by PPMD on February 21, 2018 at 1:30pm — No Comments


Staff
PTC Therapeutics Receives Formal Dispute Resolution Request Decision from the FDA's Office of New Drugs

PTC Therapeutics, Inc. announced that the Office of New Drugs of the U.S. Food and Drug Administration has reiterated the FDA's prior position and denied PTC's appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren. In its letter, the Office of New Drugs recommended a possible path forward for the ataluren NDA submission…

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Added by PPMD on February 20, 2018 at 9:00am — No Comments


Staff
End Duchenne Tour Recap: Morristown, NJ

PPMD kicked off the 2018 End Duchenne Tour with a stop in our very own backyard - Goryeb Children's Hospital in Morristown, New Jersey! PPMD was extremely grateful to partner…

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Added by Ryan Fischer on February 15, 2018 at 2:30pm — No Comments


Staff
[Webinar Recording & FAQs] Positive Data from Summit's PhaseOUT DMD Ezutromid Clinical Trial

Last week, Parent Project Muscular Dystrophy and Action Duchenne hosted a webinar update on the recent report that Summit has announced…

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Added by PPMD on February 15, 2018 at 11:30am — No Comments


Staff
FDA Publishes Finalized Duchenne Guidance for Drug Development

Today is a historic moment for the Duchenne community, as moments ago the FDA published the finalized “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment” Guidance for Industry…

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Added by PPMD on February 15, 2018 at 10:30am — No Comments


Staff
Catabasis Reports Edasalonexent Preserved Muscle Function & Substantially Slowed Duchenne Progression

Catabasis Pharmaceuticals reported new positive efficacy and safety results showing preservation of muscle function and sustained disease-modifying effects in boys with Duchene in the MoveDMD trial open-label extension following 48 and 60 weeks of treatment with edasalonexent. Consistent improvements in all assessments of muscle function were observed after more than a year of oral 100 mg/kg/day edasalonexent treatment compared to the rates of change in the…

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Added by PPMD on February 13, 2018 at 9:00am — No Comments


Staff
Action Alert! Americans with Disabilities Act (ADA) in Jeopardy

The Americans with Disabilities Act (ADA) is in jeopardy and a current bill in the House of Representatives threatens to undermine the protections of the ADA.



We need all members of our Duchenne community to contact your…

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Added by Annie Kennedy on February 12, 2018 at 9:30am — No Comments


Staff
Action Alert! Americans with Disabilities Act (ADA) in Jeopardy



UPDATE - FEBRUARY 20, 2018:
 Despite bold outcry from disability and civil rights groups, HR 620 has passed in the House. PPMD has been in touch with Senate leadership and the path in the Senate is uncertain, but we urge our community to…

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Added by Annie Kennedy on February 12, 2018 at 9:30am — No Comments


Staff
From our Hearts to Yours — Updates in Duchenne Cardiac Care Guidelines

Care for the heart has long been a priority for PPMD. Over the past several years, PPMD’s Cardiac Initiative has given rise to:



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Added by Kathi Kinnett on February 7, 2018 at 1:31pm — No Comments


Staff
Santhera Launches U.S. Expanded Access Program with Idebenone for People with Duchenne

Santhera Pharmaceuticals announced the launch of a U.S. Expanded Access Program (EAP) referred to as BreatheDMD with idebenone for people with Duchenne. Through the BreatheDMD program, eligible patients in the U.S. with Duchenne who are 10 years and older and in respiratory function decline, can obtain access to…

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Added by PPMD on February 6, 2018 at 8:00am — No Comments


Staff
Summit Therapeutics Shares Interim 24-Week Data from PhaseOut DMD Clinical Trial

Today, Summit announced positive 24-week interim data from PhaseOut DMD, their Phase 2 clinical trial of the utrophin modulator ezutromid.…

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Added by PPMD on January 25, 2018 at 10:00am — No Comments


Staff
Updated Care Consideration Guidelines for Duchenne Published

PPMD is thrilled to announce that after many years in development, the Centers for Disease Control (CDC) has published updated Care Considerations for Duchenne in The Lancet…

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Added by Kathi Kinnett on January 24, 2018 at 10:30am — No Comments


Staff
PPMD's 2018 Advocacy Conference | Schedule of Events

The PPMD Advocacy Conference registration is live! Join us March 4-6 in Washington DC to advocate for Duchenne…

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Added by Ryan Fischer on January 23, 2018 at 4:50pm — No Comments


Staff
Meet the 2018 PPMD Adult Advisory Committee (PAAC)

In January of 2014 we held our inaugural PAAC Leadership Workshop in Washington,DC. The day included engagement with federal policy makers, congressional staff, and research leaders. At the end of the day, a long-time colleague of mine from the FDA who had spent the entire day with us pulled me aside…

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Added by Annie Kennedy on January 17, 2018 at 10:30am — 2 Comments


Staff
First Duchenne Patient Dosed in Microdystrophin Gene Therapy!

(Photo used with permission from family.)



“DMD gene therapy went well. It was started at 1:15 PM and ended at 2:27 PM”    



On Thursday, January 4, I received this message from Dr. Jerry Mendell accompanied by the photo above – Dr. Mendell at…

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Added by Pat Furlong on January 10, 2018 at 10:00am — 3 Comments


Staff
2018 Off to a Strong Start — 'Patient Experience Data’ Section Added to FDA Reviews

Remember that hot June day in 2015 when hundreds of Duchenne community members rallied together at the U.S. Capitol for PPMD’s EndDuchenne rally? That same day, our longtime Congressional champions Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) introduced the Patient Focused Impact…

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Added by Annie Kennedy on January 9, 2018 at 3:30pm — No Comments


Staff
PPMD's Certified Duchenne Care Center Program – 2017 Impact & Progress

PPMD's Certified Duchenne Care Center (CDCC) Program was developed to make comprehensive Duchenne care and services, provided in agreement with the…

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Added by Kathi Kinnett on January 8, 2018 at 4:30pm — No Comments


Staff
How You Can Report Serious Drug Side Effects to the FDA

When our children are hurting, we go to our medicine cabinets and take great care to select products that will ease their pain. Reduce the fever. Relieve the ache.



In Duchenne, we know that the products and supplements prescribed often come with side effects. But we carefully weigh…

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Added by Pat Furlong on January 3, 2018 at 12:00pm — No Comments


Staff
We did it! Because of your commitment & courage!



Parent Project Muscular Dystrophy believes in the promise that gene therapy holds for the Duchenne community. And so we set an ambitious fundraising goal for our Gene Therapy Initiative. In fact, $500,000 is our largest fundraising goal ever.



And once…

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Added by PPMD on January 3, 2018 at 9:30am — No Comments

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