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April 2016 Blog Posts (15)

CRISPR/Cas9 Gene Editing

We finally have some very promising news for the development of adequate treatment for our boys with mutations requiring double or multi exon skipping!

In reading through this article about injecting a fully functional version of the dystrophin gene into the body using a harmless virus, it doesn't mention anything about applicability of multi-…

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Added by Jeffrey Kopp on April 27, 2016 at 3:37pm — No Comments


Staff
Changing the Paradigm: The Eteplirsen Ad Comm

There is no way around it, today was hard. Hundreds of us – the largest gathering of Duchenne families in history – came together to tell the FDA that eteplirsen is safe, effective, and should be approved using the tools…

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Added by Pat Furlong on April 25, 2016 at 10:30pm — 5 Comments


Staff
April 25: FDA Advisory Committee Meeting for Eteplirsen - Live Webcast Information

On Monday, April 25, an FDA Advisory Committee will review eteplirsen for potential approval and use within our Duchenne community.

The Duchenne community is joining forces in a show of strength to demonstrate to the FDA our community’s support for our robust therapeutic…

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Added by PPMD on April 21, 2016 at 8:30am — No Comments


Staff
Briefing Documents for Rescheduled Eteplirsen Ad Comm Released

The briefing documents for Sarepta’s Ad Comm on Monday, April 25th have been released. Like you, we…

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Added by PPMD on April 21, 2016 at 8:00am — No Comments


Staff
PPMD Advocates Ensure Congress Recognizes Importance of Flexibility in FDA Reviews

All your calls, emails, and Hill visits during our February Advocacy Conference (in the pouring rain!) are yielding GROUND BREAKING RESULTS.

 …

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Added by PPMD on April 19, 2016 at 4:00pm — No Comments


Staff
PPMD Names Children’s Hospital Los Angeles 10th Certified Duchenne Care Center

PPMD Awards Tenth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care

Children’s Hospital Los Angeles (CHLA) became the tenth clinic named a Certified Duchenne Care Center by…

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Added by PPMD on April 19, 2016 at 9:00am — No Comments


Staff
PPMD’s #EverySingleOne Tour Launched in Baltimore!

On Saturday, Duchenne community members from six states gathered together at the beautiful facilities at Kennedy Krieger in Baltimore – one of PPMD’s Certified Duchenne Care Centers – to hear…

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Added by Annie Kennedy on April 18, 2016 at 12:30pm — No Comments


Staff
Senate Sends Strong Letter to FDA about Duchenne Therapies

Bipartisan Group of Senators Urge FDA to Utilize All Available Tools to Speed Access to Promising Duchenne Therapies

 

On Friday April…

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Added by Ryan Fischer on April 18, 2016 at 9:44am — No Comments


Staff
Parent Project Muscular Dystrophy Convenes Pulmonary Outcomes Workshop



Parent Project Muscular Dystrophy (PPMD) will convene a Pulmonary Outcomes Workshop in Bethesda, Maryland April 14-15. Leading physicians in…

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Added by PPMD on April 14, 2016 at 2:26pm — No Comments


Staff
PPMD’s Patient Preference Study about Pulmonary Outcomes – What we learned and why it matters

Late in 2015, Parent Project Muscular Dystrophy (PPMD) and our research partners at Johns Hopkins Bloomberg School of Public Health (JHSPH) released the results of…

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Added by PPMD on April 13, 2016 at 4:00pm — No Comments


Staff
Catabasis Pharmaceuticals Initiates Part B of the MoveDMD Trial of CAT-1004 for Duchenne

Catabasis announced positive biomarker data from Part A of the MoveDMD trial, as well as initiation of dosing for the first patient for Part B (Phase 2) - a 12-week trial to assess the efficacy of CAT-1004 in Duchenne. We look forward to continued success from our partners at…

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Added by PPMD on April 12, 2016 at 9:30am — No Comments


Staff
PPMD's 2016 Connect Conference Agenda Now Available!

PPMD is excited to unveil the agenda for the 22nd Annual Connect Conference in Orlando, June…

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Added by PPMD on April 11, 2016 at 1:16pm — No Comments


Staff
ACTION ALERT: Please urge Your Senators to Sign Letter to FDA – Applying FDASIA Tools to Duchenne

Earlier this year, 109 members of the House of Representatives sent a powerful letter to the FDA urging them to “utilize all available tools, resources, and authorities” in reviewing a treatment for Duchenne muscular dystrophy. Congressional champions Congressmen Mike Fitzpatrick (R-PA),…

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Added by Ryan Fischer on April 5, 2016 at 11:30am — No Comments


Staff
How You Can Help with the Upcoming Ad Comm for Eteplirsen – Every Single Voice Matters!

We are just a few short weeks from the FDA Advisory Committee Meeting for eteplirsen on April 25th. PPMD is part of a united coalition of Duchenne organizations (MakeDuchenneHistory Coalition) working together to ensure we make the…

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Added by Ryan Fischer on April 4, 2016 at 10:30am — 1 Comment

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