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All Blog Posts (1,773)


Staff
PPMD Signs Letter to Save CDC

PPMD joined 153 members of the CDC Coalition and other supporting state and national organizations in urging Congressional leaders to provide at least $7.8 billion for the Centers for Disease Control and Prevention’s programs as part of the final FY 2018 Labor, Health and…

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Added by PPMD on December 7, 2017 at 12:20pm — No Comments


Staff
Expanding PPMD’s Gene Therapy Initiative

This holiday season, PPMD is asking you to support our ongoing Gene Therapy Initiative. Gene therapy has dominated the headlines this year…

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Added by Pat Furlong on December 7, 2017 at 10:30am — No Comments


Staff
Duchenne/Becker and FSH Muscular Dystrophies Receive ICD-10 Codes

Parent Project Muscular Dystrophy, FSH Society Lead Effort to Obtain

Critical Diagnostic Classification Standard

 

Parent…

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Added by PPMD on December 7, 2017 at 8:30am — No Comments


Staff
PPMD Designates UCSF Benioff Children's Hospital San Francisco a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named UCSF Benioff Children’s Hospital San Francisco (UCSF) our 18th Certified Duchenne Care…

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Added by PPMD on December 6, 2017 at 9:30am — No Comments


Staff
Making Duchenne Care and Clinical Trials Better – Globally

An email from a mom in war-torn Baghdad who doesn’t know how to even start to seek care for her son with a new diagnosis.



A mom in India who can’t find a provider who will give her son steroids, but instead say stem cells will cure…

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Added by Kathi Kinnett on December 5, 2017 at 11:55am — No Comments


Staff
Take Action - We Can Still Impact the Tax Reform Bill

The Senate passed their tax reform bill. In the dead of night. Overnight -- shortly before 2 AM on Saturday.



Despite protests and outcries from almost every sector: non-profits, disability communities, healthcare providers, educators, universities, researchers, graduate students (future researchers, geneticists, nurses,…

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Added by Annie Kennedy on December 5, 2017 at 9:00am — No Comments


Staff
Invest in gene therapy--all gifts matched!

So much has changed in the Duchenne research landscape since we were founded back in 1994, but your commitment has been a constant since our story began.



You’ve never stopped fighting, and the promise for our community has never been greater, with recent breakthroughs in gene therapy,…

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Added by Pat Furlong on December 4, 2017 at 10:30am — No Comments


Staff
PPMD Research Update: Gene Therapy, Accelerating Clinical Trials & More

Throughout the history of Duchenne, there have been moments that we recognize as inflection points, times when we saw a significant shift in the fight to end Duchenne, when the…

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Added by Abby Bronson on November 30, 2017 at 6:30pm — No Comments


Staff
Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Duchenne

Solid Biosciences announced today that it has initiated clinical trial activities for SGT-001, the company’s lead microdystrophin gene transfer candidate for the treatment of Duchenne.



The Phase I/II adaptive study, called IGNITE DMD, will evaluate the safety and efficacy of a single intravenous (IV) dose of SGT-001 in ambulatory and non-ambulatory adolescents and children with Duchenne. Enrollment will begin at the first clinical trial site in the United…

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Added by PPMD on November 30, 2017 at 10:24am — No Comments


Staff
Capricor Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application for CAP-1002

Capricor Therapeutics today announced that the FDA has cleared its Investigational New Drug (IND) application to conduct a new clinical trial of CAP-1002, its lead investigational therapy, in boys and young men in advanced stages of Duchenne. PPMD looks forward to being join by Capricor for a community webinar tomorrow, November 30 at 1:00 PM EST…

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Added by PPMD on November 29, 2017 at 8:30pm — No Comments


Staff
PPMD’s #EverySingleOne Tour: Albuquerque, NM

On November 4, PPMD had the pleasure of working with the parents and providers of New Mexico to present our final Every Single One Tour stop of 2017 (click here for a…

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Added by Kathi Kinnett on November 20, 2017 at 9:00pm — No Comments


Staff
VIDEO: Fundamentals of Access to Duchenne Therapies

Navigating access to approved therapies can be both time consuming and daunting. The time between approval by FDA to the day of a patient’s first commercial dose can vary depending on which insurance you have, your age…

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Added by Ryan Fischer on November 20, 2017 at 8:00pm — No Comments


Staff
PPMD Signs Letter to Congressional Leaders re: National Defense Authorization Act (NDAA) Language

Earlier this month, PPMD and other advocacy organizations joined 180 U.S. Representatives and 54 U.S. Senators to request the removal of language included in the…
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Added by Annie Kennedy on November 20, 2017 at 6:30pm — No Comments


Staff
Updates in Duchenne Newborn Screening Efforts

Earlier this month, I testified on behalf of PPMD and the national Duchenne community before the Advisory Committee on Heritable Disorders in Newborns and Children – the U.S. federal…

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Added by Annie Kennedy on November 20, 2017 at 6:30pm — No Comments


Staff
Thankful for your strength and courage

What an odd year it has been. I won’t say it has been a bad year, not entirely, but it has been dark. And I don’t think it’s just me.



Even social media, something I turn to for connection, a familiar friend, a warm embrace…has felt toxic. So many Facebook…

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Added by Pat Furlong on November 16, 2017 at 1:00pm — No Comments


Staff
Capricor Reports Significant Improvements in Duchenne Patients Treated with CAP-1002 Investigational Cell Therapy

PPMD is excited to learn that Capricor's investigational cell therapy, CAP-1002, is showing significant improvements in people with Duchenne. We look forward to learning more details about this therapy in a community webinar on Wednesday, November 29 at 1pm eastern. Details coming soon!

Read the release from…

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Added by PPMD on November 15, 2017 at 2:30pm — 1 Comment


Staff
Action Alert: If Passed, Proposed Tax Plans from Both Houses Will Affect Duchenne Community

Call & Email Your Senators Today!

Both the House & Senate have now released their Tax Reform proposals, each with variations of similar policy impacts and changes that we feel an obligation to alert you to. It is anticipated that both Chambers will move this legislation swiftly and work to complete their votes during the week…

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Added by Annie Kennedy on November 15, 2017 at 1:46pm — No Comments


Staff
PPMD Submits Community Sign-On Letter to FDA Supporting Approval of Ataluren

PPMD has been continuing to engage with both PTC and the FDA on behalf of the patient community regarding the regulatory review of ataluren.



Earlier this month, …

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Added by PPMD on November 13, 2017 at 1:00pm — No Comments

HT-100

Any updates on HT-100?

Added by Moein on November 12, 2017 at 11:09pm — No Comments


Staff
Anthem Revises Policy for EXONDYS 51, Deemed Medically Necessary for Ambulatory Patients

Progress! Today, Anthem issued a revised policy regarding Sarepta’s EXONDYS 51. In the revision, Anthem declared EXONDYS 51 medically necessary for the treatment of Duchenne muscular dystrophy when all of the following criteria are met:

  1. Individual has a confirmed diagnosis of Duchenne muscular dystrophy; and
  2. Individual must be ambulatory (with or without needing an…
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Added by Annie Kennedy on November 9, 2017 at 9:58am — No Comments

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