Short bowel syndrome, which is also called short gut, occurs as a result of someone having at least half of their small intestine removed, and sometimes the removal of a portion of…
For those of us who aren't familiar with Tardive dyskinesia, here's a little background: Tardive dyskinesia is actually something like a severe, often long-lasting side effect to drugs that treat…
It all started when Romy felt that her health was growing worse after having back surgery. She believed that something was wrong, but her doctors assure her the surgery was successful.…
Joe Beernink has a blog describing his experience with Guillain-Barré Syndrome (GBS). More than three years ago, he wrote about his recovery and his healing process. He was told that the recovery…
Researchers have reported an early victory in treating sickle cell anemia with a new gene therapy. This could possibly lead to a cure. According to the U.S. National Heart, Lung, and…
In the past, spinal muscular atrophy (SMA) has typically been considered a fatal disease. But there is new hope for children with the genetic disease. In Ohio, a baby with…
When you have an immunodeficiency disease, you’re very much at risk of getting an opportunistic infection because your immune system isn’t “scanning” your body’s cells to kill off abnormal or…
Jim and Kristen Farrell have two children, Braeden and Kernan, both of whom have a progressive muscle-wasting disorder called spinal muscular atrophy (SMA). The disorder can take away a child’s…
A new way of looking at an old idea--sometimes it’s all you need for innovation. A change in perspective can have such a big effect on a study’s progress, especially…
The HCU Network of America is not only trying to spread awareness and encourage research for homocystinuria, they were founded to serve as a resource for those currently living with…
In support of Dravet syndrome, Annabel Hughes and her son Henry, will be climbing Ben Nevis on June 17—Britain’s tallest mountain. The Hughes family has been directly affected by this…
A year after 4-year-old Lyla was diagnosed with microscopic polyangiitis (MPA) in 2015, the disease became so advanced that she was placed on dialysis for 12 hours a day. Her…
When it comes to the topic of severe fatty liver disease there is good news and bad news. In the spirit of leaving on a good note, let’s get to…
Pregnancy complications can arise no matter how healthy the expectant mother is or how careful she's been to protect her precious bundle of joy. Certain complications are minor—while others pose…
When you have a bleeding disorder, you have to be careful doing everyday tasks. It changes your normal routine. It changes your life. For Malaika, her diagnosis of hypoprothrombinemia took…
Want some more news, events and announcements on Narcolepsy? We got 'em! [one_half] [/one_half] [one_half_last] A Narcolepsy Miracle Drug? First Patient In Phase 3 Study Evaluating Treatment for…
Picture this: A desperately ill man with myelodysplastic syndrome in his seventies is fighting for his very life. A beautiful young woman in a foreign country is going about her everyday life.…
Have you ever heard of “ex vivo” gene therapy? If you haven't, it means removing some cells, modifying them, and putting them back in. The goal? Curing the diseases that…
Want some more news, events and announcements on Gaucher disease? We got 'em! [one_half] [/one_half] [one_half_last] Promising Molecule Identified as Possible Treatment for Gaucher, Parkinson's Diseases NICE Changes Mind…
You’ve heard the saying, it takes a village to raise a child? Well, as much as the fiercely independent may chafe at the suggestion, it’s true: raising, teaching, and protecting…
The Neurodegeneration with Brain Iron Accumulation (NBIA) Disorders Association was established in 1996 to serve and support all those affected by PKAN, PLAN, MPAN, BPAN, and CoPAN. Of these, PKAN or…
Bronchopulmonary dysplasia, or BPD, is a chronic lung disease that most commonly occurs in premature infants and is caused by a reaction to being on mechanical ventilation, or from being on…
I think most people experience eye problems at one point or another. Maybe your child came home with "pink eye," and before long, everyone in the household had itchy, burning…
These days Frank drives around to help others with trigeminal neuralgia. He seeks to raise awareness while comforting those who are living with the condition. Before Frank was diagnosed, he…
Addilynne is a 3-year-old with living with a disease that affects her muscle movement and strength: spinal muscular atrophy (SMA). But Amanda, her mother, states that SMA is an acronym for…
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