Last week, science continued to break boundaries and save lives. In this case, a new gene therapy was used by doctors in Europe to replace the decaying skin of a…
When a baby is born prematurely, things can get complicated pretty fast. The world is a challenging place for a child whose body isn't quite equipped to function on its…
Indianapolis is known for its speedway, but this weekend, scientists, physicians and advocates for advancements in cystic fibrosis (CF) therapies are gathering there for the 31st Annual North American Cystic…
An ongoing clinical trial called ARROW is making advances in the treatment of people with relapsed and refractory multiple myeloma. Patients who took a dose of a drug called Kyprolis (carfilzomib)…
A recent study, published in the Journal of Neurology, Neuroscience & Psychiatry, suggests that diets heavy in fat can increase the risk of relapse in children with multiple sclerosis (MS).…
According to a press release put out by PTC Therapeutics, Inc., the Office of Drug Evaluation of the Food and Drug Administration on Wednesday, Oct. 25th, 2017, sent a complete…
Earlier this month, PBS released the "The Gene Doctors." The hour-long documentary follows the recent emergence of gene therapy as a novel method of treating rare diseases. The film discusses…
There's a new treatment for X-Linked Hypophosphatemia (XLH) that may be available in the not-so-distant future. XLH occurs when the kidneys process phosphate abnormally, leading to excess phosphate loss in…
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