Featured Blog Posts – March 2009 Archive (4)

Dr. Jeffrey Towbin

Cincinnati Children’s Hospital has a new member of their neuromuscular team. It is Jeffrey Towbin, MD. Dr. Towbin has been interested in and treating Duchenne patients for over 20 years. His expertise is cardiology.

We had been planning to meet for several months, but could not get our schedules aligned. During his transition from Texas, he had been coming to Cincinnati at least one week/month and finally, it is official. He is now in charge of Pediatric Cardiology. This is one of… Continue

Added by Pat Furlong on March 30, 2009 at 11:57am — 4 Comments

Newborn Screening in Duchenne

Next week I will be taking part in a meeting on newborn screening. Duchenne is a candidate for screening. I would be thankful for your opinions about newborn screening. Please do not think I am asking you to say anything about your son or to retrace your steps. I’m asking about going forward. What are your thoughts? Concerns? If you were having a baby, would you agree to newborn screening? Would you want to know? Why?

To review:

Newborn screening is the practice of… Continue

Added by Pat Furlong on March 14, 2009 at 2:10pm — 33 Comments

Identifying and prioritizing therapeutic targets for Duchenne

Who determines what drug(s) should be tested in the clinic and what are the ‘rules’ for making decisions about trials?

PPMD has requested Treat-NMD organize this committee to evaluate the therapeutic potential of approved drugs with preclinical data and develop a priority list for clinical trials. There is an immediate need to develop an impartial committee with the capability and expertise to evaluate the case for moving these drugs toward clinical trial. Evaluation must be… Continue

Added by Pat Furlong on March 9, 2009 at 2:23pm — 2 Comments

My Favorite Drug

A few weeks ago, I received a call asking about ‘my favorite drug’ for clinical trial. My answer is pretty simple. There are a number of publications suggesting certain approved drugs may have benefit in Duchenne and they run the gamut from anti-inflammatory agents to well, I cannot think of something that starts with a Z, but you get the picture.

I don’t have a “favorite.” Rather, I think you have to be thoughtful and act; do a literature review, asking questions related to the… Continue

Added by Pat Furlong on March 3, 2009 at 2:39pm — No Comments

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