February 2013 Blog Posts (18)

Rare Disease Day 2013

This year marks the sixth annual Rare Disease Day. This is an international event with various organizations planning awareness-raising activities. PPMD is excited to be honoring this important day by participating in a few different panels and workshops where Duchenne will be given a moment in the…


Added by Will Nolan on February 28, 2013 at 11:00am — No Comments

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

need to let it out

guess for me that finding out dezi had dmd was ok... it didnt really bother me i just saw him as my son nothing different maybe it was cuz he is the 7th boy in our family to have it so it was no surprize... i do have good days and bad days more good then bad .....with dmd and work and go through a divorce and re marry to someone that is so much help in our lives .... thanks for listening

Added by Leah stevens on February 18, 2013 at 3:17pm — No Comments

Enhanced MDA Clinical Pages

In June 2012, PPMD sponsored the first Transforming Duchenne Care meeting. The meeting was organized in response to clinical care discrepancies that exist between centers treating patients with Duchenne/Becker muscular dystrophy in North America. The purpose of this…


Added by Kathi Kinnett on February 14, 2013 at 4:12pm — 1 Comment

Summit forms Advisory Board to support development of Duchenne program

Summit recently announced the formation of an Advisory Board to support the scientific and clinical development of its utrophin modulator program for the treatment of Duchenne.

The Board brings together six world-renowned scientists and clinicians with expertise in the field of neuromuscular diseases, and particularly Duchenne.

Included are…


Added by PPMD on February 14, 2013 at 10:00am — 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

My Voice: Tayjus Surampudi, 17 years old

Over the last few years, we have seen something rather phenomenal happen... Our Annual Advocacy Conference use to be made up of parents and relatives of people with Duchenne. But every year, more and more people with Duchenne are joining us on Capitol Hill to tell their own story. Tayjus Surampudi, a seventeen year old from New Jersey, joined us with his parents in D.C. for…


Added by Will Nolan on February 13, 2013 at 3:00pm — No Comments

Oxygen and Duchenne

Emergencies always happen when you are away from home, after “office hours,” or over a holiday. Due to this helpful timetable, it is likely that, when an emergency occurs, patients will be seen by professionals who have no knowledge of them or of their (rare) disease. During a respiratory emergency, the first reaction of emergency personnel is to give oxygen. Giving oxygen, without proper monitoring, can be life threatening to a person with Duchenne. Several such emergencies happened this…


Added by Kathi Kinnett on February 12, 2013 at 11:00am — No Comments

Our growing family

We have really been busy around our house in the last several months.


Our daughter, Leigha, 18, started college, 


Anthony turned 16 and loves video games


Travis 13, ran cross country and joined REC basketball


Brady 8, found his love of books and all things literary,


Wyatt 4, started pre-K and is working on all the skills to get ready for Kindergarten 


Baby Carter 23 months, started playing "living…


Added by Wyatt's Mommy, Melissa on February 9, 2013 at 9:00pm — 2 Comments

Finger pointing exercise

Other than Downton Abbey, TV is not my favorite sport. On occasion, I have watched CSI, The Closer, Bones, but, truth be told, they are all the same. Person murdered, investigation, fingers point one way or another, murderer confesses. End of story.  


When my boys were diagnosed with Duchenne, I fell into the same trap. Who is responsible? Both my parents smoked, so it’s their fault? My father’s company made doctor bags (Schell Leather Goods). As a child, I would ride to the…


Added by Pat Furlong on February 7, 2013 at 10:11am — 1 Comment

Unconditional love between siblings.

You can read the blog here - http://www.sweetsaraya.com/2013/02/04/february-4-2013-ill-look-after-you/

You can read the blog here -…


Added by Sharyn Thompson on February 4, 2013 at 9:30pm — 1 Comment

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

How Wheelchair Van Conversions Work

In case you didn’t know, wheelchair vans aren’t built as wheelchair vans.  Chrysler, Dodge, Ford, and Volkswagen do not have wheelchair van departments that design and construct handicap accessible vans.  So how are you able to purchase a wheelchair van?

Well, companies like Rollx Vans convert standard vans into wheelchair accessible vans.

It’s much more than bolting a ramp on here or adding some tie-downs there.  Van…


Added by Jordan Valley on February 4, 2013 at 12:24pm — 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

Prosensa Achieves Orphan Drug Status on its Entire Duchenne Portfolio

Leiden, The Netherlands  29 January 2013 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, announced it has achieved orphan drug designation in the EU and the US for all of its compounds for the treatment of Duchenne muscular dystrophy (DMD).


The European Medicines Agency (EMA) assigned orphan drug status to Prosensa’s two preclinical compounds PRO052 and PRO055 and the US Food and…


Added by PPMD on February 1, 2013 at 12:16pm — No Comments

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