One of our partner organizations, the MDS Foundation, is hosting another free MDS Patient & Family Forum in Chapel Hill, NC. When: Saturday, March 28, 2020 Where:
Roche has recently released the results of the latest phases of their study of gantenerumab, which is a treatment of a rare form of Alzheimer’s
Prevail Therapeutics has recently been granted the Orphan Drug designation by the FDA for their gene therapy PR001, which is intended to treat Gaucher disease.
I recently had the pleasure of being interviewed by Sunshine Mugrabi about how I transformed my life over the past 20 years in the face
According to a story from Financial Buzz, the gene therapy company AVROBIO recently presented updated data on February 10th, 2020 at the 16th Annual WORLDSymposium being held
Hypotonia Ataxia, and Delayed Development Syndrome Hypotonia, Ataxia, and Delayed Development Syndrome (HADDS) is caused by mutations in the EBF3 gene which is found on
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