Featured Blog Posts – February 2013 Archive (7)

Early morning calls

The phone rings at 6AM. Something else happens inside, a feeling that something is horribly wrong. This morning, the window of my blackberry said “BRIAN D” – Brian Denger. I imagined Brian running through the snow, nails in his sneakers to cut through the ice on the streets in Biddeford Maine.


Brian’s voice, normally calm and controlled, faltered and…


Added by Pat Furlong on February 26, 2013 at 10:42am — 14 Comments

Pipeline Update: Drisapersen (GSK2402968) - GSK

GSK recently announced in their Q4 2012 results that the…


Added by PPMD on February 20, 2013 at 12:00pm — No Comments

LAST CALL: Phase 1 Survey - Risk/Benefit in Duchenne Therapies

By now you’ve probably heard about PPMD’s Risk/Benefit in Duchenne Therapies program, which aims to inform the FDA and other government agencies, biopharmaceutical companies, and others about the treatment priorities and risk tolerance of the Duchenne community. We’ve had a great response to our first survey, with more than 90 participants so…


Added by Holly Peay on February 19, 2013 at 1:56pm — 1 Comment

Advocates in Muscular Dystrophy Community Press Congress for Fast Action on Muscle Disease Research and Treatment

The Muscular Dystrophy Association, Parent Project Muscular Dystrophy and the Foundation to Eradicate Duchenne have called on the U.S. Senate and the U.S. House of Representatives to reauthorize the MD CARE Act of 2001, and to continue federal support for the accelerated pace of research and treatment development for muscular dystrophy. We are urging the public to contact their U.S. Senators and ask them to vote for reauthorization of …


Added by PPMD on February 19, 2013 at 10:30am — No Comments

Risk Tolerance, Benefit Preferences, and Treatment Priorities

The Duchenne community has entered a new era where more clinical trials are coming online and the possibility of treatment is becoming a reality. Upon completion of a trial, the FDA weighs the evidence presented. But at the end of the day, it is often a value judgment about potential benefits and risks to the patient.

PPMD has launched a program – Benefit/Risk in Duchenne Therapies – to help inform the FDA and other government agencies, biopharmaceutical…


Added by Holly Peay on February 13, 2013 at 3:30pm — 2 Comments

A Strong Start to 2013

Thanks to everyone who contributed so generously to PPMD’s holiday appeal we have been able to fund three new projects designed to speed the progress of the clinical trials underway in Duchenne. As many of you know, we have a…


Added by Sharon Hesterlee on February 4, 2013 at 2:30pm — No Comments

PPMD Funds Collection and Analysis of Imaging Data from Eteplirsen Study

Parent Project Muscular Dystrophy (PPMD) announced that it has funded $59,000 to Krista Vandenborne, PhD  of the University of Florida in Gainesville to test the ability of magnetic resonance imaging  (MRI) to detect changes in the muscles of  those living with …


Added by PPMD on February 4, 2013 at 8:30am — No Comments

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