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Treating DMD requires a mutlifaceted approach - restoring the missing protein, minimizing inflammation, minimizing fibrosis, promoting muscle growth, and restoring blood flow to affected muscle. In our crew's latest manuscript, led by Abhi Batra, he explores the use of sildenafil in mdx and wild type mice after an acute bout of downhill running and during a progressive low intensity treadmill running program. His findings reveal that PDE5 inhibition reduces muscle damage after single bouts of exercise and improves functional capacity during enduring training in mice affected by DMD.
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Dystrophin, traditionally thought of as the structural scaffolding of muscle, plays numerous metabolic roles to maintain the integrity of the sarcolemmal membrane. In Brittany's latest paper in NMR in Biomedicine, she explores age dependent changes to the metabolic profile and lipid saturation within dystrophic muscle across different ages of the disease. She demonstrates differences in healthy versus affected mice, and different metabolic changes and lipid saturation within mice as they become progressively affected by the disease. This study reveals exciting new targets for therapy, as well as further cements non-invasive quantitative imaging as a robust, safe, and effective biomarker!
M.D.-Ph.D.'s are an unusual breed. Through combining the skillsets of scientist and clinician, trainees face a unique and rigorous journey to earn the dual degrees. My recent commentary in the Medical Science Educator highlights the complementary nature of both degrees through my own experiences and provides advice to younger trainees.
“We are like dwarfs on the shoulders of giants, so that we can see more than they, and things at a greater distance, not by virtue of any sharpness of sight on our part, or any physical distinction, but because we are carried high and raised up by their giant size.” - Bernard of Chartres, circa 1159 This past Saturday, I finally graduated from the University of Florida’s College of Medicine as a Doctor in Medicine. The convoluted journey has had many peaks and troughs, providing me some of the most meaningful and most difficult times of my life. Through my own wits, I would not be in this position. Growing up, I was never the top of my class, not the most athletic, nor strongest in anything I’ve done. It is through those around me - family, friends, and mentors - who have elevated me to a position to succeed beyond my own capabilities. My family, from a non-medical background, provided me with the love and support to continue pushing through difficult times. My friends helped me celebrate the best of times and picked me up in the worst of times throughout life. My mentors, who saw a diamond in the (very) rough, pushed me to achieve outcomes that were not fathomable in my own mind. Medical school offered a real privilege to develop intimate relationships with patients and families during their most vulnerable of times. From babies to burr holes and cesareans to seizures, medical school provided a unique opportunity to experience all facets of medicine and healthcare. Besides adding more letters to my last name, nothing transformative happened as I crossed the stage to receive my diploma. I’m still the same person I was moments before, though the walk across stage symbolically represents the passage into being a physician. Though I have a whole new set of responsibilities and obligations, I still feel like the same as before. Traditionally, the principle of medicine has been to treat the patient. However, I challenge us to ask ourselves how we really “treat” patients? Treating patients goes far beyond providing prescriptions and scheduling follow up visits. It is about addressing people’s biggest fears and concerns; it is about providing compassion and empathy to people who may not otherwise feel cared for. I look forward to this challenge as residency looms around the corner, and hope to leave a lasting positive impression to those who allow me to be their healthcare provider during their most vulnerable moments of life. Though late by a few weeks because of other life events, Rosha and I are thrilled to be shipping up to Boston for my residency this June! There isn’t a better city for the both of us in terms of professional opportunities in terms of my training at Boston Children’s Hospital, and the plethora of opportunities available to Rosha in the biotech / pharm industry that Boston has to offer. It has been one chaotic ride these past few months, between our first wedding in Nepal, a second wedding in America, and a conference in Berlin, it has been one heckuva interview season. It was a real treat to be able to travel and have the chance to interview at several places, seeing how different programs are structured and function. While my thoughts were that any outcome was a good decision, as it would allow me to do what I wanted, we are quite ecstatic about the chance to move to Boston for the betterment of both of our careers. With the stress and chaos of Match behind us, my advice to rising M4s about to embark on the terrifying journey of match is to: trust your instinct, enjoy the process, realize what matters most to you, Trust your instinct. The bulk of residency training is going to be the same between institutions and it really is your colleagues that make/break your training experience. Initially, I tried making all sorts of complicated excel spreadsheets and algorithms ranking different variables between programs (research opportunities, city life, primary vs. specialty training, etc…). After a few interviews, I realized that these differences were rather subtle between programs, and that ultimately, the best indicator of programs was my voice’s excitement of my phone calls to my wife after the interviews. Anywhere you go, you end up as a well trained physician. Enjoy the process. My general rule of thumb was to not rush into/out of interviews, and to stay a day before and after the interviews to tour the city and neighborhoods around. Granted, this meant sleeping on someone’s couch an extra day or two, but frankly, to me it was really important to tour the city to see where I am going to be calling home for the next 5 years. Also, the Match interviews are entirely different than Medical School interviews. Where, in medical school, you just want “someone to take you!” However, in the match interviews, the roles are flipped, as programs realize they have many qualified candidates and go out of their way to sell their program to you, wanting you to come to their program. Certainly, you must appropriately prepare for the interviews, but letting your personality shine through can really go a long way in the interview process. It’s a strange sensation feeling wanted, but something definitely worth enjoying for a change. What matters most: Perhaps most importantly, realizing what is most important to you as you embark on the next personal and professional chapter of your life is probably the most important factor influencing the match decision. Are family and location most important? Are you geographically focused on a particular area for any number of reasons, or conversely, do you want to explore somewhere new and exciting? What else matters in your rank list: reputation, camaraderie, or subspecialty or primary focuses? Asking these sort of questions for yourself will help you identify which institution and city will help you become the best version of yourself. As archaic and convoluted as it may seem, the match process ultimately is best attempt to make the process as organized and fair as possible. Though, for many Type A’s of medical school, the lack of control is intimidating and daunting, the match process ultimately works out best for just about everyone involved! With much dividing discussion surrounding the debates about guns and their associated violence, I took the opportunity to share my thoughts and opinions in the Gainesville Sun through an Op-Ed. In response to Rick Scott signing the first gun law in 30 years, I talk about how guns are a part of the American DNA, and how this is not necessarily a good thing. Though well intended gun owners intend to prevent death and harm with their guns, there are 34 criminal homicides, 78 gun suicides, and 2 accidental deaths for every crime prevented or justifiable homicide. Furthermore, there is bipartisan support for common sense gun laws, something that evades current governmental legislators. In response to the Parkland shooting, we, as Floridians, have a chance to lay the groundwork for a slow and long overhaul of the current gun laws to provide safety to our citizens. After all, which is more important: the right to gun ownership, or the right to health and safety?
In the March 2018 Florida Chapter of the American Academy of Pediatrics, we published a brief article to better understand and provide information about the troubles that non-citizens, particularly children, face in this country.
Healthcare for Non-Citizen Children What is a non-citizen?
Who are we talking about?
Why does this matter?
Where do you get healthcare as a non-citizen?
Health consequences to immigrant children
Where do we go from here?
Resources for medical students and healthcare professionals: https://www.nilc.org/issues/health-care/ https://www.healthcare.gov/blog/10-things-immigrant-families-need-to-know-about-the-marketplace/ Resources for pediatric patients and their families: https://www.healthcare.gov/immigrants/coverage/ https://www.floridakidcare.org/eligibility/ Resources to get involved with policymakers https://www.senate.gov/reference/common/faq/How_to_correspond_senators.htm https://www.house.gov/representatives/find-your-representative 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. |
