PPMD Community

All Blog Posts Tagged 'PPMD Blog' (713)


Staff
Deciphering What We Know About EMFLAZA

The unknowns. Some of the hardest beasts to conquer in life.

 

The unpredictable variables that make life impossible to plan.

 

The not being able to commit to vacations and holiday plans because – well, you just don’t know what curve balls life will toss at you between…

Continue

Added by Pat Furlong on May 15, 2017 at 9:30am — 1 Comment


Staff
In Honor of Moms: A Note from Tom Furlong

This Mother’s Day, thank someone for their role in the fight to end Duchenne.

If you’re reading this blog, chances are you know my wife, Pat Furlong. For 23 years now, Pat has been leading Parent Project Muscular Dystrophy (PPMD) and the fight to end Duchenne. Many of you have told me over the years that Pat has been a source of comfort,…

Continue

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


Staff
PPMD's 2017 Connect Conference Agenda is Now Available!

PPMD is excited to unveil the agenda for our 2017 Connect Conference in Chicago, June 29 - July 2, 2017!

 

We are so proud of the …

Continue

Added by PPMD on May 9, 2017 at 11:00am — No Comments


Staff
PPMD's Response to PTC Announcement of EMFLAZA (deflazacort) Pricing & Access

Today, PTC Therapeutics provided a community update on EMFLAZA (deflazacort) which they acquired earlier in the year from Marathon Pharmaceuticals. The community has anxiously awaited an update on both access and pricing ever since the deal between PTC and Marathon was announced.



On today’s call, we…

Continue

Added by PPMD on May 8, 2017 at 5:38pm — No Comments


Staff
PTC Therapeutics to Discuss Launch Plans for Emflaza™ (Deflazacort) during Community Call on Monday, May 8th at 3:00 PM EDT

PTC Therapeutics has released a letter to the community and will host a conference call for the Duchenne community to discuss the launch plans for EMFLAZA™ (deflazacort) on Monday, May 8, 2017 at 3:00 PM EDT (call-in details are below).



On a…

Continue

Added by PPMD on May 8, 2017 at 10:00am — 1 Comment


Staff
URGENT Action Alert – Call Congress Now Before This Afternoon’s AHCA Vote!

The newest version of the GOP HealthCare Bill is coming to a vote by 1:30 PM EDT this afternoon!

This is moving fast and we need ALL MEMBERS of our community to …

Continue

Added by Ryan Fischer on May 4, 2017 at 12:00pm — No Comments


Staff
ACTION ALERT: ­Immediate Action Needed – Revised AHCA Bill Threatens Duchenne Community’s Access to Health Care Coverage

The current version of American Health Care Act (AHCA) directly threatens essential health benefits (EHBs), Medicaid eligibility and funding, and vital health care protections for our Duchenne community. 

 

I wish there was a way to…

Continue

Added by Annie Kennedy on April 28, 2017 at 9:30am — No Comments


Staff
PTC Therapeutics Announces Community Conference Call on May 8 to Discuss Launch Plans for Emflaza™ (Deflazacort)

PTC Therapeutics, Inc. will host a conference call for the Duchenne community to discuss the launch plans for Emflaza™ (deflazacort) on Monday, May 8, 2017 at…

Continue

Added by PPMD on April 26, 2017 at 10:00am — No Comments


Staff
Capricor Therapeutics Announces Positive Six-Month Results from the Randomized Phase I/II HOPE Clinical Trial in Duchenne Muscular Dystrophy

Capricor Therapeutics today announced positive top-line results from a safety and exploratory efficacy analysis of six-month data from the randomized 12-month Phase I/II HOPE Clinical Trial of CAP-1002 (allogeneic cardiosphere-derived cells), an investigational candidate for the treatment of patients with Duchenne.



We are excited to see these results and to see the field of cell…

Continue

Added by PPMD on April 25, 2017 at 10:24am — 2 Comments


Staff
FDA Announces Pediatric Advisory Committee Meeting on May 18

On May 18, 2017, FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s…

Continue

Added by PPMD on April 24, 2017 at 12:30pm — No Comments


Staff
PTC Therapeutics Completes Acquisition of Emflaza™ for the Treatment of Duchenne in the U.S.

Today, PTC Therapeutics announced that they have completed their acquisition of Emflaza™ (deflazacort) ahead of schedule, following early conclusion of the anti-trust review.



PTC will be hosting a call with the Duchenne community the week of May 8 to provide details on the plan for the launch of Emflaza. We look forward to learning more from PTC in the…

Continue

Added by PPMD on April 20, 2017 at 4:00pm — No Comments


Staff
PPMD Convenes Meeting to Explore the Burden of Pediatric Clinical Trials

The Duchenne community has waited 20 years to be where we are. We are reminded daily of how far we have come, how the landscape has changed, how there are close to 20 clinical trials to choose from. While these changes within are landscape bring us to a hopeful time in Duchenne, the journey to…

Continue

Added by Kathi Kinnett on April 20, 2017 at 12:30pm — No Comments


Staff
Members of Congress Re-Introduce ABLE Improvement Bills

Great news – The ABLE program enhancements have been re-introduced in Congress and are gaining support. This package of bills include improvements to the original ABLE program that directly impact our community, here’s how:



On April 4th, a bi-partisan group of Members of Congress,…

Continue

Added by Annie Kennedy on April 14, 2017 at 9:00pm — No Comments


Staff
PPMD Joins Letter to Protect Medicaid

In late March, PPMD joined 87 organizations to issue a letter calling on Congress to take a hard look at the likely significant and…

Continue

Added by Annie Kennedy on April 13, 2017 at 9:30pm — No Comments


Staff
PPMD Signs Appropriation Letter to Fund Newborn Screening

PPMD is proud to sign on to letters sent both to the Senate and the House asking that they provide at least $29.8 million to the Centers for Disease Control and Prevention’s (CDC) Newborn Screening Quality Assurance Program (NSQAP) and at least $19.9 million to the Health Resources and Services…

Continue

Added by Annie Kennedy on April 12, 2017 at 9:00pm — No Comments


Staff
PPMD’s #EverySingleOne Tour: Iowa City, IA

Iowa Children’s Hospital marked our third stop on PPMD’s 2017 Every Single One Tour, held on April 1, 2017. The amazing staff at Iowa Children’s Hospital helped make this a wonderful day and we can see…

Continue

Added by PPMD on April 7, 2017 at 1:30pm — No Comments


Staff
PPMD Designates UC Davis a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named UC Davis Department of Physical Medicine and Rehabilitation Neuromuscular Disease Clinic (UC…

Continue

Added by PPMD on April 6, 2017 at 12:30pm — No Comments


Staff
PPMD's Duchenne Drug Development Roundtable Meeting Series, Part 3: Clinical Trial Readiness from the Viewpoint of Clinicians and Infrastructure

Accelerating trials. Protecting our robust therapeutic pipeline. Ensure as many hits on goal as we can.



Priorities shared by all of us.

And to achieve this, PPMD has convened our industry partners through our Duchenne Drug Development Roundtable…

Continue

Added by Annie Kennedy on April 5, 2017 at 11:00am — No Comments


Staff
PPMD Working with FDA to Plan Landmark Pediatric Advisory Committee Meeting to Improve Clinical Trial Experience

Later this spring – likely on or about May 18 – FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s Essence trial.

Once again, our community is at a pivotal moment in our history where we…

Continue

Added by Annie Kennedy on April 5, 2017 at 10:00am — No Comments


Staff
PPMD’s #EverySingleOne Tour: Seattle, WA

The PPMD Every Single [One] Tour had an amazing turnout last weekend in Seattle, WA! Nearly 100 parents, patients and professionals gathered at Seattle Children’s Hospital to talk and learn about research, care, therapy, and clinical…

Continue

Added by PPMD on March 31, 2017 at 11:30am — No Comments

Monthly Archives

2017

2016

2015

2014

2013

2012

2011

2010

2009

2008

2007

© 2017   Created by PPMD.   Powered by

Badges  |  Report an Issue  |  Privacy Policy  |  Terms of Service