PPMD Community

All Blog Posts (1,641)


Staff
ATTENTION: All Patients on Masters’ Deflazacort

As you know, the FDA is currently reviewing Marathon Pharmaceutical’s New Drug Application (NDA) for deflazacort for the treatment of Duchenne. It…

Continue

Added by PPMD on January 16, 2017 at 6:30pm — No Comments


Staff
New Resource to Help Navigate EXONDYS 51 Access

Prior to the approval of EXONDYS 51™, we knew that access to emerging therapies would be a process – and an often challenging process at…

Continue

Added by Annie Kennedy on January 13, 2017 at 11:00am — No Comments


Staff
New Research & Clinical Trial Tools on PPMD's DuchenneConnect!

We are excited to announce three new features now available on PPMD's DuchenneConnect! These tools are meant to help you in your journey as you gather information around potential research studies and potential new exon…

Continue

Added by Ann Lucas on January 12, 2017 at 1:24pm — No Comments


Staff
Akashi Therapeutics Provides Update on Three Duchenne Compounds



This weekend, Akashi Therapeutics announced they are working toward having three novel, complementary compounds in the clinic in 2017 with potential to treat all Duchenne patients independent of their specific genetic…

Continue

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


Staff
Sarepta Therapeutics Enters into Research Agreement and Option Agreement with Nationwide Children’s Hospital for Microdystrophin Gene Therapy Program

On the heels of PPMD’s announcement last week of a $2.2 million grant to Nationwide Children’s Hospital’s gene therapy study being led by Dr. Jerry Mendell and Dr. Louise Rodino-Klapac, Sarepta Therapeutics today announced their commitment to the trial through a separate research agreement with Nationwide Children’s, including an…

Continue

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


Staff
PTC Provides Update on Next Steps for Translarna

In a corporate update released today, PTC Therapeutics announced next steps for their drug, Translarna in the U.S. According to the release, PTC plans to file the Translarna New Drug Application (NDA) for nmDMD over protest with the U.S. FDA in…

Continue

Added by PPMD on January 9, 2017 at 10:30am — No Comments


Staff
PPMD Awards Nationwide Children’s Hospital $2.2 Million Grant to Explore Gene Therapy in Duchenne

Today, PPMD announced a $2.2 million dollar grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PI), and Nationwide Children’s Hospital.  Our purpose in awarding this grant is to open and accelerate the field of gene therapy – a strategy that seemed impossible as a potential…

Continue

Added by Pat Furlong on January 6, 2017 at 9:00am — No Comments


Staff
Two simple words...THANK YOU!

We not only met our goal, we surpassed it!





We could not be more overwhelmed by the incredible response to our holiday campaign. In many ways, with a therapeutic pipeline full of promising treatments, it feels like the future of Duchenne therapy is here. Thanks to this community, we head into 2017 with…

Continue

Added by Pat Furlong on January 4, 2017 at 3:47pm — No Comments


Staff
What You Need to Know about Duchenne & Viral Gastroenteritis (That Horrible Stomach Bug!)

Viral gastrointestinal (GI) viruses are no fun for anyone, but they are especially worrisome for a person living with Duchenne muscular dystrophy. GI viruses affect the GI track – the stomach and intestine (i.e., ‘gut’) – resulting in abdominal pain/discomfort, nausea, vomiting, intestinal…

Continue

Added by Kathi Kinnett on January 4, 2017 at 10:30am — No Comments


Staff
Make my parents pay--48 hours left!

We’re so close to our goal to fund CRISPR/Cas9!…

Continue

Added by PPMD on December 29, 2016 at 9:30am — No Comments


Staff
This could help me--and everyone with Duchenne

Your donation will be DOUBLED. Now is the moment to give.…

Continue

Added by PPMD on December 22, 2016 at 9:12am — No Comments


Staff
DOUBLE your investment in the future

Help us raise $250,000 by 12/31!…



Continue

Added by Abby Bronson on December 20, 2016 at 12:00pm — No Comments


Staff
Understanding the Path to Access – Resources for the Community

We have all had frustrations getting healthcare paid for, whether it is getting access and coverage for appointments, procedures, equipment, and/or medications.

Coverage is especially difficult when new medicines or procedures are recommended. Parents,…

Continue

Added by PPMD on December 19, 2016 at 12:30pm — 1 Comment


Staff
I Will Match Your Gift

Help us raise $250,000 to fund CRISPR/Cas9.



After a few months of relative quiet, noise has returned to the Killian house in full force! For the holidays, all five kids are back under one roof with a revolving door of friends and endless groceries. Even with all the chaos that entails, this is my favorite time of…

Continue

Added by PPMD on December 13, 2016 at 9:38am — No Comments


Staff
New Paper Further Validates Need for Annual Cardiac MRI Surveillance in Duchenne

When PPMD convened the Contemporary Issues in Duchenne Cardiology meeting with National Heart, Lung, and Blood Institute (NHLBI) in July 2014, there were several recommendations.

The strongest recommendations included the use of cardiac…

Continue

Added by Kathi Kinnett on December 8, 2016 at 4:00pm — No Comments


Staff
PPMD Is Seizing This Moment to Explore the Potential of CRISPR/Cas9 in Duchenne

It is the holidays – time for giving and laughter and hope. Hope comes in many forms, but I would imagine that for many reading this blog, hope comes in the form of a cure for Duchenne.  

Could CRISPR/Cas9 offer hope for treating Duchenne? 

As background, CRISPR/Cas9 is a…

Continue

Added by Abby Bronson on December 8, 2016 at 3:30pm — No Comments


Staff
How PPMD’s Certified Duchenne Care Centers Are Improving Quality & Access to Care

When I joined PPMD in 2012, care for Duchenne was all over the map. 

 

Sadly, there were over 150 clinics spread across the US, but there was no oversight of these clinics – the community did not know who was providing care, the level of care being provided or whether anyone at…

Continue

Added by Kathi Kinnett on December 8, 2016 at 3:02pm — No Comments


Staff
BREAKING NEWS: 21st Century Cures Crossed Over the Finish Line!

The Duchenne Community’s Voice Was Essential Every Step of the Way & Contains PPMD’s Provision to Strengthen Patient Focused Drug Development Efforts



To everyone in our PPMD community who called, reached out, emailed, and met with your elected…

Continue

Added by Annie Kennedy on December 7, 2016 at 2:30pm — No Comments


Staff
PPMD’s #EverySingleOne Tour: Durham

Duke University marked our final stop on PPMD’s 2016 Every Single One Tour, held on November 2. We were thrilled to be holding another tour stop at one of…

Continue

Added by Ryan Fischer on December 7, 2016 at 11:04am — No Comments


Staff
Light up the Lines!! Urge Your Senators to Vote YES on 21st Century Cures!

We need you to call your two US Senators TODAY with one simple message – VOTE YES ON 21st CENTURY CURES!!

 

With just days remaining in the current Congress, this bill represents the extraordinary efforts of our PPMD community’s work and outreach with…

Continue

Added by Ryan Fischer on December 5, 2016 at 10:07am — 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