Each year just under 1,500 infants are born with spina bifida, a name given to a cluster of malformations that affect the neural tube. Of those 1,500, only 43%…
According to a story from USA Today, Thanksgiving 2009 was a harrowing time for the Anderson family. About a month earlier, Darrin and Sarah Anderson's oldest son Henry had died…
Status Update: Shared Decision-Making in Hematology Sunday, December 1, 2019 Beth Faiman, CNP, PhD Certified nurse practitioner at the Cleveland Clinic in Cleveland, Ohio In this edition, ASH Clinical News…
Global Blood Therapeutics was recently granted priority review by the FDA for their new drug Oxbryta. This drug is meant to treat sickle cell disease, and it is the…
According to a story from biopharmadive.com, the field of gene therapy has gained increasing relevance throughout 2019, and 2020 will likely be a continuation of this trend. An area of…
According to a story from Myeloma Research News, the first patient has been dosed with an experimental allogeneic (donor derived) CAR-T cell therapy called UCARTCS1 as part of a phase…
Finding the correct doctor for treatment can be difficult, especially if one has a rare disease. Because few doctors specialize in rare diseases, they are often spread across the…
One thing many rare patients know is that many rare diseases do not currently have any therapies. However, that is changing. There are so many possible solutions that need coordination,…
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