Terrence Merrill shares the challenging emotional realities of raising a child with Sanfilippo syndrome. You can read more background about the Merrill's battle against this rare disease in our article here.…
At the age of 28, Suzy remembers medical students feeling her puffy hands and feet. Her feet were so expanded she had to wear wide shoe sizes. Her fingers were…
When I was diagnosed with Parkinson's Disease (PD) in 2000, I first thought of my three children and how my diagnosis would impact them. How would I tell them? Would I…
12-year-old Cezy Fosca lives in Kilkenny City and suffers from Morquio syndrome. Her family waited for months on approval for her treatment, after she lost access to it. Finally, the…
When people are faced with cancer, their main concerns are understandably treatment and survival. However, many are shocked to discover that when they survive cancer, another sinister blow on their…
A gene therapy called ABO-202 that is being developed to treat Batten disease has just been granted Orphan Drug Designation by the European Medicines Agency (EMA), reports BioPortfolio. This follows…
The phrase “it takes a village” gets tossed around a lot. But in Codicote, that’s what’s happening. Fundraising events and challenges are being held left and right. All of them…
The US Food and Drug Administration (FDA) has just approved the drug Vonvendi for use in adult patients with von Willebrand disease who are undergoing surgery. This is an extension…
Imagine not being able to eat because you cannot swallow your food. Add to that intolerable pain and the inability to sleep at night. These are some of the symptoms…
In order to honor Cystinosis Awareness Day, which takes place on May 7th each year, a man will attempt to walk 57 miles in a single day in order to…
What if paranoia could be genetically located? A study of Prader-Willi syndrome gave scientists a chance to find out. Keep reading to learn more about this development, or follow the…
In St. Louis, Missouri, a local start-up is using novel research to make a big difference. The start-up, known as Unleash seeks to develop a unique cure for cancer. Keep…
We write a lot about rare diseases that affect people but what about the rare diseases among animals? If you own a horse, you've likely heard of Equine Infectious Anemia…
A new device to treat incontinence is being developed by Peter Sayet at Precision Medical Devices Inc., reports the Orlando Sentinel. Incontinence is the term used when people pass urine…
Happy Friday Everybody! This week we're spotlighting two parts of one patient story. We also have news on Alfie Evans and epilepsy research. Sit back and enjoy this week's Editor's…
A Phase 1 clinical trial on patients with cancers that are the result of a change to the tyrosine kinase receptor, also called RET, has produced encouraging results. Cancers caused…
Researchers at the University of Hong Kong have discovered that a drug used to treat muscular dystrophy also works on a hereditary heart disease called Lamin A/C-related dilated cardiomyopathy. The…
A group of researchers led by Professor Gholson Lyon has discovered a genetic mutation that is linked to certain traits, reports Medical Xpress. The method used to discover it was…
According to a story from Neuroscience News, researchers have begun to test the effectiveness of a certain molecule in fighting against childhood brain cancers, such as medulloblastoma. It has the…
A healthy diet and a good exercise regimen go a long way towards maintaining good health. When health care professionals recommend this combination they’re usually trying to keep dangerous diseases…
Richard Kuhns (B.S.Ch.E) boasts twenty years experience working with Morgellons disease and its sufferers. Others consider him to be a leading expert on skin parasites, and the complications of Lyme…
On April 16th, global healthcare company Grifols announced its donation of 25 million international units (IU) of blood clotting factor drugs. These donations occurred within the last year, and are…
I lost my fiancé, Ashley “Bea” Briggs, due to complications from cystic fibrosis a little over a year ago in early 2017. We met early in the summer of 2015…
The prolonged legal battle over Alfie Evans, a toddler with a serious undiagnosed degenerative neurological disease, has reached a final verdict. The UK doctors currently caring for Alfie made the…
The US Food and Drug Administration (FDA) has just approved a new drug called Tavalisse™ to treat adults who have thrombocytopenia as a result of chronic immune thrombocytopenia (ITP), following…
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