Neuropsychiatric disorders can be some of the most challenging conditions to understand. Mechanically, socially, and biologically, it is often difficult to make sense of their innermost workings. Recent research, however,…
Pharmaceutical company and gene therapy leader, uniQure, published promising data from their current phase I/II trial testing out a gene therapy called AMT-060 in patients with serious cases of hemophilia B.…
Kei Otake is a 19-year-old cello student at The Juilliard School - a prestigious performing arts college in New York City that boasts alumni like Viola Davis, Robin Williams, Adam Driver…
A five-year-old boy who spent the majority of his life battling neuroblastoma can now rest in peace. Jacob Jones passed away at a hospice with his mother and father sitting…
The Washington Post published a bizarre story earlier this month that is equal parts interesting and terrifying! A US National Security Agency counterintelligence officer (AKA a spy) is claiming that while…
If you've been waiting for a new rheumatoid arthritis treatment, good news is on the way! Rheumatoid arthritis is a chronic inflammatory condition that causes joint pain among other symptoms.…
Why are US children with cystic fibrosis improving faster than Canadian kids? Newborn screenings and child healthcare programs may be behind the improvement among US children with cystic fibrosis. There's…
A new study may lead to new cures and treatments for Marfan syndrome. Researchers at Johns Hopkins have made great progress testing in mice. Follow the original story here. On…
Gene therapy has been on the horizon for years now, but before today, it was only a reality for patients with non-inherited diseases caused by genetic mutations. That just changed!…
5-year-old Gia Danninger was diagnosed with aplastic anemia last year. Ever since, it's been a constant battle for her life, but Gia fights with courage. This rare blood disorder causes…
After dueling with a fatal rare disease, Makenna Vanzant, will be making her epic return to the basketball court. The high school sophomore has been battling hemolytic uremic syndrome and…
What do Nathaniel, a child with Bardet-Biedl Syndrome and Sylvester Stallone's famous boxing hero have in common? Keep reading below, or follow the original story to find out! Braille reading…
On this season of America's Got Talent, the world met Christian Guardino. I was one of millions who heard his beautiful singing voice as he belted out his song onstage.…
Patients diagnosed with Duchenne muscular dystrophy (DMD) have always lived with a grim prognosis. Many don't make it into their 20s and face debilitating symptoms years earlier-- a dark reminder…
One in 10,000 babies is affected by CHARGE Syndrome. Anabelle is one of them. According to doctors, Anabelle should be deaf and blind. She should need to be fed through…
Jay and Amy Granzow are among the many worried parents fearing the GOP tax plan, because of the threat it brings to orphan drug research. The Manhattan Beach couple fear…
Robbie Edwards has a rare disease, one so rare that she's one of only 17 people in the world with it. She suffers from hereditary spastic paraplegia which represents a…
As a scientist, Mary-Claire King has had a remarkable career related to her study of BRCA genes. She’s received a Lasker Award and has been recognized by President Obama with…
Not all bacteria are created equal, but all play a role in sustaining--or breaking down--health. And when you're talking about children with cystic fibrosis (CF), a lung disease that impairs…
Allison and Ben Gauvin have been to hell and back, but they manage to keep their spirits up. The couple from Connecticut lost their two children to a rare mitochondrial…
The internet has been trying to figure out the caffeine question for years: is it good for us? Is it bad for us? Will it stunt our growth? Will it prevent…
Happy Holidays, Patient Worthians! As we celebrate the holidays this year, we take a moment to reflect on the things that matter most deeply to us. This week, we have…
In the UK, Newcastle University and Pfizer are collaborating on a $39.9 million-dollar funded project aimed at developing greater tools for testing patients at the most risk for non-alcoholic fatty…
Although Parkinson's disease (PD) affects men and women equally, researchers have discovered an area of disparity: care giving. A study published this month in Neurology by researchers at the Perelman School of Medicine…
Xander Williamson is among 600 people in the world, and merely 11 people in Australia, to have Pitt-Hopkins syndrome. His mother Melissa had no idea she would be returning from…
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