About five months ago, Lexi Robins was born in the United Kingdom. She didn't show any signs of abnormal development at first, besides the fact that her thumb didn't move…
According to a recent news release from biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx"), the company's therapy Mepsevii (vestronidase alfa) was approved in Spain for reimbursement. The entire reimbursement process takes…
As reported in Times Union; this was a year where the numbers of many contagious diseases saw steep declines as the world stepped up in handwashing and mask-wearing. In the…
According to the Cancer Network, orelabrutinib, a Bruton tyrosine kinase (BTK) inhibitor, was recently granted Breakthrough Therapy designation within the United States. While the treatment is also indicated for patients…
According to a story from MarketWatch, the biotechnology company Cyclo Therapeutics announced that is has proceeded with the enrollment for its phase 3 clinical trial. This trial will evaluate an…
What do phenylketonuria (PKU), galactosemia, and maple syrup urine disease (MSUD) have in common? All three are considered inborn errors of metabolism, or rare genetic disorders in which the…
In a review posted on dovepress.com, monoclonal antibodies have established themselves as an important tool in the management of multiple sclerosis in both the relapsing-remitting form and the progressive form.…
Ally Winter was diagnosed with short bowel syndrome when she was four years old. Now, she is 17 years old and working to raise awareness for rare and chronic illnesses.…
Happy Friday! This week, we examine the struggle to access experimental rare therapies through compassionate use. Also, in our new Compassion Corner series, we examine why addressing patients' personal wellbeing can…
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