Who said helping cure and treat rare diseases couldn't be fun?! Last week, Three Lake Partners - a venture firm that invests in healthcare and quality-of-life solutions for patients dealing…
In a recent study, researchers at Stanford University School of Medicine successfully cured mice of cancer. Already, patients diagnosed with lymphoma are being sought out to undergo clinical trials with…
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…
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!…
Shantee Anaquod has gone from planning the end of her life to planning the rest of her life. After being bed-ridden for over a month with the fatal disease, atypical…
Sangamo Therapeutics shared some big news about the search for Mucopolysaccharidosis II (MPS II) treatment. They reported that they have, for the very first time, used experimental in vivo gene therapy to treat…
My son Nick has a rare, debilitating, life threatening disease called Lennox-Gastaut Syndrome (LGS). To learn more about LGS, click here. For over 38 years I have been consciously aware…
Crohn’s disease may affect as many as 1.6 million Americans, according to the Crohn's & Colitis Foundation (CCF). Although it can strike at any age, Crohn's is more prevalent among…
Jessica Gilbert uses her rare neurological disorder as fuel for her creativity. The Converse College student suffers from misophonia, which translates into "the hatred of sound." People who suffer from…
Next time you order up fries, you might want to think twice before sprinkling on the sodium chloride—but not for the obvious health reasons. Sodium is a no-no for people…
Cielo Honasan has Beckwith-Widemann Syndrome and is an amazing athlete. She's been running since she was little. She never let her disability slow her down. Read on to learn more…
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…
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…
It’s always exciting when two giants of their fields join together to accomplish something. When Pixar and Disney joined forces to bring us the best animated films since… ever, the…
If you're looking for a time table for adjusting to, and accepting, the diagnosis of a rare disease, you won't find one, because there isn't one. The variety of rare…
Multiple Sclerosis is a disease of the brain and spinal cord. In patients with MS, their immune system attacks the protective myelin sheath that usually covers nerve fibers. These nerve…
Although there are treatments currently available with a proven track record against rare types of cancers, such as Waldenstrom macroglobulinemia, with any treatment, however successful, there will be a handful…
Klippel-Feil Syndrome Awareness Day began five years ago, when a small group of people decided a specific day each year should be set aside, to help the KFS community speak…
Let’s talk clinical trials. Clinical trials are part of clinical research and at the heart of all medical advances. Clinical trials look at new ways to prevent, detect, or treat…
A phase 3 clinical trial has produced positive results for cutaneous T-cell lymphoma (CTCL) patients. The drug tested is called brentuximab vedotin (Adcetris). Specifically, the drug was created to treat…
According to CBC, Marie-Eve Chainey was on dialysis six out of seven days a week. For 15 years, this was her life. It was a necessary feat to control her…
The Associated Professional Sleep Societies held their 31st annual meeting in Boston June 3rd through 7th. You can read more about Sleep 2017 by clicking here. Among other discussions was cataplexy.…
On June 21st, Targeted Oncology posted the results from a phase 2 clinical trial for chronic lymphocytic leukemia (CLL). The trial examined the effects of combing two therapies--obinutuzumab and bendamustine.…
Due to a rare disease affecting many parts of her body, my oldest daughter, Kelley, saw a number of different doctors in a number of different locations. I clearly remember…
Scientists aren’t always the best at sharing amongst themselves. But sharing data could mean accelerated research. By making your own data available to scientists you can help contribute to a…
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