A recent article in CheckOrphan describes a study that was conducted by Researchers at the Lysosomal and Rare Disorders Research and Treatment Center in Vancouver, B.C. The study examines the…
Proteus Syndrome Proteus syndrome is a rare disease caused by the overgrowth of tissues including the skin, adipose tissue, and parts of the central nervous system. It also affects the…
Alexion Pharmaceuticals, Inc., a company that is advancing a rare disease pipeline in complement biology, recently issued a series of press releases announcing positive results of a Phase 3 study…
This is the story of five-year-old Aubree Fox who has recently been diagnosed with hydrocephalus, stenosis, and an aneurysm in her brain. This slew of life-threatening diagnoses were all a mystery…
According to a story from Gene42, a new rare disease platform called Genomics4RD has been launched in Canada. This is the first national research platform dedicated to rare disease to…
Ofatumumab as a Single Agent Results of ofatumumab administered as a single agent with an extended induction (initial treatment) brought about a high response rate and it was well tolerated among follicular…
NICE stands for the National Institute for Health and Care Excellence in England. This organization is responsible for evaluating the cost effectiveness of all medications as they come to the…
“There Is No Gene for the Human Spirit”—from the movie Gattaca, 1997 Whether it’s distant uncles purported to be horse thieves in the early days of the…
The treatment of metastatic renal cell carcinoma (RCC) has evolved over the last ten years and investigators now look towards combination therapies to improve response rates and durability for…
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