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November 2017 Blog Posts (14)


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 — No Comments


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


Staff
"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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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


Staff
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…

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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…

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


Staff
Update on Formal Dispute Resolution Process For Ataluren

On October 25, PTC Therapeutics shared the news that the FDA had issued a Complete Response Letter regarding their application for ataluren, noting that FDA had…

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Added by Annie Kennedy on November 2, 2017 at 9:30am — No Comments

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