Sometimes it takes a tragedy to create real change. Last year, Connecticut joined the short list of states to implement an ALD newborn screening test, after the parents of a…
Researchers who study rare diseases often look at what's going wrong in the body in order to find a treatment. But the team studying rare lung diseases at the Perelman School…
Most women take for granted their ability to become pregnant and bring new life into the world -- until they can't. For more than seven million U.S. women with polycystic…
Some diseases are a mystery. If the name of the disorder starts with "idiopathic," it means that no one really knows what causes it. That's the case with a rare…
In the ultimate feat of recycling, scientists at UCLA and the Howard Hughes Medical Institute have re-purposed the LeXis gene as a cholesterol-lowering agent. A recent press release issued by…
When my son was eight years old he joined the swim team. For two hours a day, five days a week, he splashed around the pool mastering the Australian Crawl…
Over the years, I've talked to dozens of people with Parkinson's disease (PD). They come from all socio-economic backgrounds. Rich and poor. Male and female. But the one thing they…
How can you inspire the world to be more heart-healthy--even if you have a rare disease such as pulmonary arterial hypertension (PAH)? That's the mission of this year's World Heart…
Nearly 100 million Americans live with chronic pain. That's a world of hurt. Chronic pain is defined as persistent pain that lasts more than three months. According to Johns Hopkins…
In 1973, the lifespan of a person with sickle cell disease (SCD) in the U.S. was only 14 years. Thanks to the dauntless efforts of researchers, scientists, and clinicians, today…
There is so much about how our bodies work that we take for granted. The careful concert of organs, systems, and structures that allow us to breathe, walk, talk, and…
The 1960s were a time of social rebellion and reform. The Civil Rights Movement, the feminist movement, and Vatican II all rose from a growing sense of dissatisfaction with the…
Chances are you grew up with the fragrant scent of baby powder perfuming the air. Your mom may have used it to prevent your diaper rash or to sooth herself…
Mantle Cell Lymphoma (MCL) is a very rare cancer of the blood. So rare in fact, that there are only 4,200 cases diagnosed in the U.S. each year and…
Odds are someone you know has a chronic, even debilitating illness, but you probably have no clue how it's affecting his/her life. Just because a condition isn't obvious doesn't mean…
The human brain is a mystery. Although enormous strides have been made to understand how it works and how our neuro pathways respond to various stimuli, there is still so…
In the world of medical science, there's theory and there's application. Many scientists and researcher make amazing discoveries, but few find applications that can really transform lives. Hans Clevers is…
In our derisive political environment, it's hard to find something everyone can agree on. But this month--Ovarian Cancer Awareness month, to be exact--party lines are coming together to heighten knowledge about…
Exciting research is happening right now that could help thousands of children around the world with cystic fibrosis (CF). A group of scientists and researchers in Australia are determined to…
Aidan Jack Seeger was a curly-headed, blonde boy with incredible blue eyes. At age seven he succumbed to a rare disease called adrenoleukodystrophy (ALD)—only 11 months after his diagnosis. Had…
Epilepsy is a general term for a variety of conditions that misfire in the brain's neuroreceptors. Before the condition was studied scientifically, people often thought that seizures were anything from…
Confession time: I was a Trekkie. Yes, I lusted after Captain Kirk. But deep down my heart belonged to Bones, the smack-talking ship's chief medical officer who often saved the…
It pays to have a friend who's a research endrocrinologist—especially when you have a rare and little-known disease like acromegaly. For Oregon-based teacher, Betsy Whispell, the friendship was a lifesaver. She…
There's a whole lot more going on in your body than you know. Right now, trillions of microorganisms are hanging out in the gut (and elsewhere,) sustained in their own…
What do you do when you have a disease called acromegaly that causes you to keep growing into a 7'1", 346-pound force of nature? If you're Dalip Singh Rana (aka…
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