October 2013 Blog Posts (14)

MDCC meeting report

The Muscular Dystrophy Coordinating Committee (MDCC) had its 10th meeting the end of this summer at the National Institutes of Health offices in Bethesda, MD.  The members of the MDCC come from federal agencies involved in one or more muscular dystrophy programs as well as public representatives from several organizations representing one or more forms of muscular dystrophy.  There are representatives from the National Institutes of Health (NINDS, NIAMS, NICHD and NHLBI), Centers…


Added by Brian Denger on October 30, 2013 at 11:50am — No Comments

Who Owns Your Clinical Trial Data?

When parents of children with Duchenne enroll their children in a clinical trial there is often a sense of participating in something bigger than themselves—of course you hope that your child may benefit from a new therapeutic approach, but you also know that all those days of missed school, waiting rooms, endless syringes, and painful biopsies are…


Added by Sharon Hesterlee on October 29, 2013 at 11:00am — No Comments

10/14 – 10/18: An Amazing Week in Review

When Chris and Patrick were diagnosed, there was no genetic testing. No therapies were targeted to the “CODE” and no way to DECODE Duchenne. We made assumptions about carrier status and lived many years with only assumptions. I thought my mother was a carrier. I knew I was a carrier. The doctor made that assumption for me. Serial CKs were done on our daughters with the…


Added by Pat Furlong on October 23, 2013 at 10:30am — No Comments

Inside a Case Study for Pediatric Heart Failure

2013 Pediatric Heart Failure Summit

When medical meetings want to present a particular topic, they introduce the topic as a “case study.” A case study is the story of a single person that incudes the topic of interest.  While the topic might be of interest to a larger group of people, the case study is an N=1, the discussion of…


Added by Kathi Kinnett on October 21, 2013 at 12:00pm — No Comments

Steroids or NOT???

We are at the stage right now that the Dr's are saying it is time for steroids for my son.  I am not sure about it.... Has anyone out there NOT given their son steroids ...and if so...how is it going?

Added by Laurie on October 18, 2013 at 6:51pm — No Comments

How to Decode Duchenne

We are excited to announce that Parent Project Muscular Dystrophy and Sarepta Therapeutics are joining forces on a genetic testing program for patients with Duchenne or Becker muscular dystrophy. Through the new genetic testing program, called Decode…


Added by Pat Furlong on October 17, 2013 at 9:00am — No Comments

My Voice: Jake Pritchard, 9 years old

When a family hosts a fundraising event for Duchenne, we are thrilled and extremely grateful. When a family hosts the same event six years in a row, we are overwhelmed! This year the Pritchard family, along with their friends – Meghan and Jason Alpern – in Matthews, North Carolina will host the…


Added by Will Nolan on October 15, 2013 at 11:30am — No Comments

Pushing the FDA to Accelerate Treatments

We all know it: rare diseases, especially progressive pediatric disorders like Duchenne, are different than common diseases. The FDA knows it too, but they don’t have a plan for how to treat them differently. They are working on different ways to weigh the benefits and risks of treatments for rare disease versus treatments for treat common…


Added by Holly Peay on October 10, 2013 at 9:30am — No Comments

NOT using Steroids...

Has anyone decided to NOT place their son on a steroid medicine???  Looking for comparisons....

Added by Laurie on October 9, 2013 at 8:22pm — No Comments

Clinical Trial Expectations

I was privileged to share some of the data PPMD has collected in our Clinical Trial Expectations study at the World Muscle Society poster session. Many clinicians and industry and academic scientists came to the poster to learn about our community thoughts and preferences, and were quite interested in how they might improve the clinical trial process. Highlighting the…


Added by Holly Peay on October 7, 2013 at 10:58am — No Comments

World Muscle Society Congress: Considering Care

Care related research was popular at this year’s 18th International World Muscle Society Congress. While this is not a complete summary, I will try to hit the highlights. 


Several poster sessions, as well as the first portion of the Sarepta Symposium, presented by Eugenio Mercuri, MD, PhD (Rome, Italy)…


Added by Kathi Kinnett on October 4, 2013 at 12:59pm — No Comments

World Muscle Society Congress: Next Generation Exon-Skipping & Clinical Trial Updates

The World Muscle Society Meeting in Monterey, California saw reports on a number of topics of interest to the community. On Thursday poster sessions covered advances in the next generation of exon-skipping and updates on various clinical trials.

Next Generation Exon-Skipping

Several laboratories presented data on…


Added by Sharon Hesterlee on October 4, 2013 at 9:30am — No Comments

On Being Diurnal

Guest post by Ivy Scherbarth. Ivy is a Colorado/Wyoming FACES Coordinator for PPMD and mom to Hazel, age 8, and Rain, age 6. Rain has Duchenne muscular dystrophy. Follow Ivy at her blog, Living …


Added by PPMD on October 4, 2013 at 9:30am — No Comments

Important Update: Flu Vaccinations for 2013-14

Flu season is back and the vaccine is slightly different this year:

The “nasal spray flu vaccine” is a live attenuated vaccine, which contains living, but weakened virus.  It is generally not recommended for people with neuromuscular diseases on or off corticosteroids.  It is safe for caretakers, siblings and classmates who are…


Added by Kathi Kinnett on October 3, 2013 at 10:37am — 2 Comments

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