Neurocutaneous syndromes are conditions that affect a person’s skin and nervous systems. They are present from birth. These disorders are incurable, but treatments are available to help manage ...
Sanfilippo syndrome, also known as mucopolysaccharidosis type III, is a rare life-threatening disorder that interferes with metabolism. While it doesn’t have a cure, some symptoms can be treated to ...
Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) ...
Hepatorenal syndrome is when the kidneys stop working properly due to liver failure. It is a life threatening condition. In most cases, a person will not survive without a liver transplant. There are ...
Taneia Surles is health and wellness writer and public health professional that has been creating health and wellness content since 2020. Her writing has covered several verticals, including sexual ...
Hunter syndrome (MPS II) presentations will include analysis from continued follow-up of Phase 1/2 data for tividenofusp alfa (DNL310), currently under FDA Priority ReviewPreliminary data from Phase 1 ...
Stickler syndrome is a rare disease of the connective tissues that mostly affects areas around the eyes, ears, face, and mouth. Retinal detachment is a common complication of this disease, and it’s ...
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