Tag Archives: rare

The most expensive medicine ever treats a rare condition. The price: $2.13 million – Los Angeles Times

Evelyn Villarreal was one of the first children treated, at 8 weeks. Her family, from Centreville, Va., had lost their first child to spinal muscular atrophy at 15 months. Two years later when Evelyn was born a test showed she also had the disease, so the family enrolled her in the gene therapy study at… Read More »

Research may predict outcomes for newborns with rare disease

Up until now, scientists have understood that there are two main subtypes of the disease- known as diffuse and focal. Diffuse CHI affects the entire pancreas, whilst focal CHI affects just one area of the organ, forming a focal lesion. The team from Manchester now understand that focal CHI can be further categorised into two types- spreading focal lesions and isolated… Read More »

Boy, six, is left in a coma after his 'headache' was a rare condition that caused his brain to SWELL

Boy, six, is left in a coma after his ‘headache and vomiting’ turned out to be a rare condition that caused his brain to SWELL up Ellis Artist woke up in the middle of the night screaming in pain last Tuesday He was rushed to hospital where doctors believed a rash was meningitis MRI scans… Read More »