Earlier in June, the U.S. Food and Drug Administration (FDA) won a major legal victory by getting an injunction to prevent Florida-based US Stem Cell Clinic from offering its treatments. The company claimed to create stem cells from patients’ body fat and use these to treat a variety of serious illnesses, including Parkinson’s disease, ALS, and chronic lung disease. This company is just one of many that have sprung up ...

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With the recent FDA approval, Zolgensma became the world’s most expensive medication. Priced at $2.125 million per patient, the one-dose gene therapy is a potential life-saver for children with spinal muscular atrophy (SMA). Now, the treatment is at the center of an intensifying debate over the rising price of medications.

Industry watchdogs are outraged. They say Zolgensma is merely the ...

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I know what kinds of things I'm supposed to tell you. I know that your other would be advisors will usually stick to the same basic messages: "Keep reaching for your dreams" or "your hard work is all worth it in the end" or "your education will lay the world at your feet." These platitudes are all well and good, but honestly, they're forgettable. And pretty words don't always translate ...

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Two recent patient experiences prompted this post. In the Wall Street Journal, Dana Hawkins-Simons described several years of being seen by specialist after specialist for her complaints of tiredness, dizziness, ringing in the ears, palpitations, shortness of breath and “brain fog.” She finally researched her symptoms and demanded that she be checked for vitamin B12 deficiency, which turned out to be the cause of her symptoms. Recently, the ...

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When I served as president of the New Jersey Neurosurgical Society, I met a lot of people from around our great state.  One question that I heard from many parents and coaches was, “After a concussion, when is it safe to let a student-athlete return to play?” It is an important question because hundreds of thousands of high school students in the USA suffer concussions annually.  I have given this question ...

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As a college freshman in 2012, my life seemed perfect. I was attending Rhodes College in Memphis, TN, with the dream of becoming a pediatric oncologist. To that end, I was serving as a volunteer, researcher, and clinical intern at the world-renowned St. Jude Children’s Research Hospital. I was thriving in my classes and building incredible relationships with my peers and professors. I had never been happier. Everything changed when I ...

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Stress! It is a fact of life for all human beings. Stress is a normal reaction — psychologic and physiologic — to the everyday demands of life. When your brain perceives a threat, the body reacts with a fight-or-flight response, releasing hormones, increasing heart rate, and blood pressure. When the situation is over, your body returns to a normal, calm state. But sometimes, in our overly-stimulated world, the stress response button ...

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It was the last week of my EMS elective. I was incredibly lucky to ride with one of the EMS captains who was eager to take me to any call that sounded interesting. We were called to a possible stroke. An 82-year-old woman with sudden-onset unilateral weakness and expressive aphasia. The paramedic and EMT on scene were two I had met before. We actually talked earlier that day about the ...

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In medical school, you’re not taught how to give stellar patient presentations. Yes, you’re shown the traditional order of things: “Give an effective one-liner first, then tell the HPI [history of present illness] but only give pertinent info, etc.” Just exactly how to deliver the punch that impresses your attending is an art. And it’s an art that takes some time to perfect. My very first time presenting a patient was ...

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As my patients aged along with me, I noticed both the expected increased numbers of people with some form of dementia and even more patients who were worried about this topic. Unless you have been away on a ten-year safari, you are very aware that dementia is a growing problem. Some 5.8 million Americans are living with Alzheimer's disease, and similar numbers occur in most western countries. The incidence goes ...

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When a patient comes to me long after he or she suspects that something is amiss, I tell them, “I understand.” I know why they had ignored their tremors for too long. I understand why they had dismissed their balance issues as insignificant. Depictions of Parkinson’s disease in popular culture are grim, and nobody wants to be told that they have a disorder that is associated with loss of motor skills ...

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Nothing quite catches our collective attention like clear and dire issues that need solutions and traumatic brain injuries (TBI), the leading cause of death and disability across all population demographics, are no exception. More and more research is demonstrating the serious and widespread impacts of TBI.  Every day, about 153 people die from injuries that include TBI. In 2013 alone, the U.S. Centers for Disease Control and Prevention ...

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Two prestigious medical journals published studies involving Alzheimer disease (AD) and amyloid beta in successive weeks; both were paired with guest expert commentaries (editorials).  One editorial, about a drug study, also reviewed several other failures in anti-amyloid therapy and stated: “To be blunt, A-beta [amyloid] lowering seems like an ineffective approach….” The other editorial had an encouraging attitude toward amyloid PET imaging as part of the diagnostic ...

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“As soon as I mentioned the 'A' word, I knew she would not be calling me back.”  This was what one of the parents I met at my preschool told me about what it was like trying to register her son with autism in a non-specialized local community program. Once the registrar heard the “A word” – autism – they said that another child had already taken the last spot available, ...

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Alex and Edward Santos have just returned from a weekend away at their aunt and uncle’s place. They love it there. The couple is in their 20s and spoil the boys with late bedtimes, movies and video games, and home-made cupcakes with chocolate icing and sprinkles. It’s been a long break for Alex, who’s 15, and has been off school for a week. Now, his vacation is almost over, he ...

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Patients should have as much access to information as possible. The more they know — the more informed decisions about their health and life that can be made. However, faulty headlines about new therapies are infiltrating news outlets left and right. Many times this information can be misleading, and it is difficult for patients, their friends, and family members to decipher. Lately, there has been a surge in the release of ...

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He struggled to breathe, progressing from deep breaths with wet sounds reverberating in his lungs, to guppy breathing — opening the mouth like a fish, contorting the entire face. His heart rate slowly decreased, from 150 beats a minute — a pace attempting to keep up with the oxygen demands of the body — to 50 beats, to 40 beats, to that which is incompatible with life. The nurse pushed ...

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I pause for a moment and close my eyes -- to center myself in this country, where I am today.   I am about to walk through the door and tell the parents what my assessment has shown. I nod to the interpreter waiting behind me and knock firmly before entering the room.  You see, I am the bringer of news, the bringer of new, unwanted knowledge that validates their fears, ...

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Parkinson’s disease (PD) is a progressive neurodegenerative disorder that affects movement, resulting most notably in tremor, slow movement, stiffness and balance issues. While Parkinson’s patients benefit from medicine that can alleviate some of these symptoms, there are no available treatments to slow, halt or reverse the progressive destruction of dopamine-producing brain cells and abnormal clumping of alpha synuclein, an otherwise normal brain protein. The disease affects roughly 10 million people ...

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“Guys, are you alright in there?” I ask casually while taking the first bite of my dinner on a 24-hour PICU shift. No answer. “What is going on? Why is she beeping so much? Is her tube blocked?” My voice gets louder. Complete human silence. Except for the deafening hypoxia monitor going to 30s. Heart rate monitor dipping to below 60s. All this beeping was coming from a six-month-old girl with a history ...

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