Last week in my blog I touched on…

In February 2013, a bipartisan group of Senators and Representatives introduced the MD-CARE Act Amendments of 2013 (S. 315 and H.R. 594). This legislation seeks to update the landmark MD-CARE Act law…

Several weeks ago, I was invited to participate in a discussion focused on how large pharmaceutical companies should communicate with advocacy; how communication needs to be different in rare disease; and more specifically what advocates in rare diseases expect, want, and need from pharmaceutical companies. I was one of…

Wading through policy is no easy task.  But imagine policy as the white lines on the road, the boundaries within which FDA operates.  Building our case, showing how Duchenne fits into those white lines and demonstrating how and when Accelerated Approval makes perfect sense is the purpose of PPMD’s white paper.   …

The following article appeared online today on InsideHealthPolicy.com, a subscription service. We are sharing the full article here.

Posted: May 16, 2013

Rare Disease Group Pushes Endpoint Discussion In FDASIA Guide

Without key changes to FDA's consideration of surrogate and intermediate endpoints, drugs in the pipeline to treat muscular dystrophy will not make it to…

We are always grateful to anyone who hosts an event, runs a race, or even sells lemonade to raise money and awareness for the fight to end Duchenne. But when a family consistently hosts a community event, that grows year after year, well...we are overwhelmed! The Knight family from Colorado will celebrate the…

Mary-Lou Weisman

Mary-Lou Weisman, author of Intensive Care and founder of Fund for Pete's Sake. To learn more or to read Mary-Lou's blog, visit …

This summer, PPMD will host a pre-conference the day before the , addressing the issue of transitioning individuals living with Duchenne from pediatrics, through adolescence and into adulthood.  We realize that this is a very complex process that will require a program that should begin as early as age 12, and will extend well into adulthood.  The purpose of the pre-conference is to provide a first step that will bring together parents, patients, providers, and policy makers to begin to…

Two weeks ago, PPMD proudly published Putting Patients First, a white paper outlining recommendations to speed responsible access to new therapies for Duchenne. In that short amount of time, the reaction to this white paper has been phenomenal. From interest by the media to accolades from industry and agencies, we have been truly humbled by the response we have received.

And while the…

Meeting Summary

Last June, just prior to the Connect Conference, PPMD hosted the first Transforming Duchenne Care meeting.  This meeting was attended by health care providers and administrators from 17 institutions, parents, and representatives from MDA, CDC, NIH, and industry.  Key themes emerged from this meeting:

1. Currently in the US, there are significant discrepancies in care for patients with Duchenne and Becker.
2. Parents would…

Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD. Follow Ivy at her blog, Living Duchenne.

 …

April 29-30 in Washington, D.C.

Recently I had the privilege of attending and participating in the Symposium on Best Practices in Clinical Study Design of Rare Diseases in Washington, D.C. The symposium was organized in collaboration with Children’s National Medical Center (CNMC), Clinical and Translational Science Institute of Children’s National (CTSI), The George Washington University, and Research in Pediatric Developmental Pharmacology Centers (RPDP).  This…

Left ventricular assist devices (LVADs) are fairly new to the pediatric population and are very new to the Duchenne community.  So new, in fact, that, in the fall of 2012, only three patients have had LVADs implanted worldwide.  As dilated cardiomyopathy and heart failure remain the major cause of death in patients with Duchenne, this novel therapy could possibly have far reaching effects.  Below are two LVAD stories, from two different perspectives: first a young man with Duchenne (Jason)…

CLINICAL TRIALS – The path to approval.

Typically we think about the Clinical Trial Process in the context of our kids.   We hear about Phase I trials.   Depending on the drug/biologic, many of these compounds will be required to be tested in healthy human subjects.   Some Phase I trials have inclusion criteria, suggesting they are looking for healthy adults of a certain age to take a single or multiple doses of a compound or biologic.   The purpose of the study…