Contrast-Enhanced Near-Infrared Optical Imaging Detects Exacerbation and Amelioration of Murine Muscular Dystrophy
In our most recent article, in Molecular Imaging, we explore the potential to use Near Infrared Optical Imaging as a biomarker to assess muscle pathology secondary to disease processes, specifically two variants of muscular dystrophy. As these diseases are progressive by nature, it is important to be able assess progression of disease. With the rapid pipeline of treatments reaching clinical trials, it was equally important to be able to assess mitigation of disease. Overall, we demonstrate the ability to utilize this technology to further assess worsening and reversal of muscle pathology in two disease states.
During the recent Imaging in Neuromuscular Disease 2017 conference held in Berlin, I took the opportunity to venture away from the conference to visit the Berlin Wall Memorial. Though constructed for different purposes, walls inevitably and always isolate and exclude. With much racist, xenophobic, and ignorantly blind patriotism permeating America's current political scene, I composed an Op-Ed, published in the Gainesville Sun, in an attempt to remind ourselves of our history to avoid repeating such a past. After all, America is the land of immigrants, and building walls is counterintuitive to our core American values.
Deflazacort, a glucocorticoid that has been used off-label for years to mitigate the disease progression of Duchenne muscular dystrophy (DMD) was recently given formal approval by the FDA for use in DMD. This formal approval requires patients and families to now purchase the drug, even if they have been inexpensively using it for years, from a single pharmaceutical company for significantly higher prices. The increased price of Deflazacort has caused much concern and burden to patient families, who previously had been using it safely for years, but are not required to purchase it for elevated prices.
Rosha and I took the opportunity to write a viewpoint in JAMA Neurology to voice our concerns over the new costs of this old medicine.
I'm a current MD-PhD candidate, working hard to help treat and manage muscular dystrophies