Good news for mantle cell lymphoma patients! A new treatment called Calquence has just received approval from the U.S. Food and Drug Administration (FDA) for adults who suffer from this rare…
According to an encouraging letter she wrote in The FCS Foundation, the day that Melissa learned that her beautiful 3-week-old daughter had a rare disease- familial chylomicronemia syndrome (FCS), or lipoprotein lipase…
As parents, we want to protect our children from harm and keep them safe. So when your child is suffering from a rare metabolic disorder, it's natural to feel helpless.…
Pauline Siegel was 35-years-old when she was unexpectedly struck with a rare disease that would render her disabled from the waist down. The diagnosis took several painful days to finally…
Rare disease research can be daunting. It's much more difficult for rare, or orphan, diseases to find adequate funding to develop treatments than it is for diseases that have larger…
Every year, families travel across town and from around the country to be together for the holidays. This year, while you're sharing pumpkin pie and Black Friday Sale flyers, take…
Sean Kisielnicki's life is ruled by Hunter syndrome. This rare condition not only affects him, but takes a toll on the entire family. The morning routine is always the same.…
November is Pulmonary Hypertension Awareness month, and this year the Pulmonary Hypertension Association (PHA) has launched a special, nation-wide campaign to raise awareness for the disease called The Right Heart. Pulmonary…
Former Jersey Boys star Jeff Leibow, organized his first Neurofibromatosis Hope benefit concert in Las Vegas in 2011. His hope was to make it an annual event that would go…
Puberty, development, and menopause - most parents have found that explaining these concepts to kids, though entirely natural and unavoidable, comes with a certain degree of discomfort. In girls in…
The Alpha-1 Foundation has a website dedicated to Alpha-1 patients and caregivers. It offers a wealth of information and resources about about the disease. Alpha-1 Antitrypsin Deficiency (Alpha-1) is a…
If you or someone you love have Friedreich's Ataxia, you know how badly we need an adequate treatment. Friedreich's Ataxia (FA) is a rare disorder, which results in neurological and…
Nothing puts "fun" in a fundraiser than holding that event at Walt Disney World. For the past 25 years, Disney has hosted an annual marathon to help support hundreds of…
Severe diarrhea. Daily abdominal pain. Extremely high triglycerides. Chronic pancreatitis. Joint pain. Incredibly uncomfortable conditions for anyone. Now imagine you’ve experienced this pain your entire life without knowing what was…
A new film coming out this month will change the way you think about Apert Syndrome and similar disease. Keep reading to learn more about the film, or follow the…
Abi Galatia suffers from stage three osteosarcoma, a very rare and very deadly form of bone cancer. The weird part is, she dreamed that she would get the disease for…
Enfermedad crónica afecta para siempre la perspectiva de la vida de un paciente. A veces, esa perspectiva depende de cómo el paciente se siente físicamente en ese momento. Después de…
Happy Friday! We have some great news for the short bowel syndrome community. We also have info on a PBS doc you won't want to miss. One of our contributors…
Let's take a trip to West Yorkshire, England, where a rare disease story is unfolding in the intense world of rugby. Rob Burrow is a retired English professor turned rugby…
Indianapolis is known for its speedway, but this weekend, scientists, physicians and advocates for advancements in cystic fibrosis (CF) therapies are gathering there for the 31st Annual North American Cystic…
Exciting updates have come out of Alnylam Pharmaceuticals. They just released data from one of their highly anticipated trials for its leading rare disease drug. It looks like it did…
Up until now, diagnosing ovarian cancer hasn't been so easy. Most of the time, it goes undiagnosed until it's already advanced. At that point, treatment is harder and less reliable.…
Dogs, with their pure hearts and boundless supply of affection, have made life better for humans for a long time. Dogs aren't just making people feel better by giving them…
Familial partial lipodystrophy (FPL) is a rare genetic disorder that causes fat cells to die off. This makes people with this disorder suffer from the progressive loss of body fat. At…
Getting a cold or flu is bad enough for most "healthy" people, but if you have a progressive, incurable lung disease, such as idiopathic pulmonary fibrosis (IPF), cystic fibrosis, COPD,…
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