November 2017 Blog Posts (21)

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…


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

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…


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

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…


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

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 preview of our 2018 Tour schedule).  We had the pleasure of…


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

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 and disease progression, and even where you live. With two…


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

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 Senate version of the National Defense Authorization Act…

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

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 advisory committee on newborn…


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

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 posts are gas soaked, with every “Like” a potential flame.…


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

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…


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

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…


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

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, PPMD reached out to FDA requesting guidance on…


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


Any updates on HT-100?

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

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…

Added by Annie Kennedy on November 9, 2017 at 9:58am — No Comments

"My Journey on the 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?

Hear first-hand what it's like to serve on the PPMD Adult Advisory Committee (PAAC) from current…


Added by Annie Kennedy on November 8, 2017 at 2:30pm — No Comments

Wave Life Sciences Initiates Clinical Trial for Lead Program in Duchenne

Wave Life Sciences Ltd. announced the initiation of a global Phase 1 clinical trial for WVE-210201 in Duchenne patients amenable to exon 51 skipping. PPMD is excited by the progress Wave Life Sciences has made in exon skipping. While advances have been made in exon skipping, especially recently, there is more to explore within this technology. We…


Added by PPMD on November 6, 2017 at 9:09am — No Comments

Sarepta & Nationwide Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the Treatment of Duchenne

More good news in gene therapy! Today, Sarepta Therapeutics announced that Nationwide Children’s Hospital has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for the micro-dystrophin gene therapy program. The Phase 1/2a clinical trial, now open to enrollment and scheduled for initiation by mid-November, is designed to assess the safety and tolerability of AAVrh74.MHCK7.micro-Dystrophin in individuals…


Added by PPMD on November 6, 2017 at 8:30am — No Comments

Action Alert: Proposed Senate Tax Plan & the Direct Impact on Our Community

Call & Email Your Senators Today!

Yesterday, the Senate released the Tax Cuts and Jobs Act which includes many issues that we feel an obligation to alert you to.


While PPMD does not typically engage in legislation related to tax reform, this proposal (being referred to as…


Added by Annie Kennedy on November 3, 2017 at 10:00am — No Comments

Sarepta Therapeutics & Nationwide Children’s Hospital Announce FDA Clearance of the IND Application for the GALGT2 Gene Therapy Program

Once again, PPMD is excited to see industry, patient groups, and research partnering, as today Nationwide Children's Hospital and Sarepta Therapeutics announced the achievement of an important milestone in…


Added by PPMD on November 3, 2017 at 8:39am — No Comments

SMA Gene Therapy Study: Their Success is Our Hope

Yesterday, the New England Journal of Medicine (NEJM) published “Single-Dose Gene-Replacement Therapy for Spinal Muscle Atrophy.” 

Spinal muscular atrophy (SMA) is a disorder caused by a mutation in the survival motor neuron gene 1…


Added by Pat Furlong on November 2, 2017 at 9:54pm — No Comments

My Journey on the PAAC

I decided to join the PAAC after Parent Project’s 2015 Annual Connect conference in Washington, D.C. Annie, along with the 2015 PAAC members and a few other adults with Duchenne, hosted a pre-conference meeting for teens and adults living with Duchenne. When listening to the other adults like me speak I felt a strong connection and saw myself fitting right in up…


Added by Colin Werth on November 2, 2017 at 2:21pm — No Comments

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