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 ...
Children with Down syndrome have a significantly increased risk of leukemia, while adults have a lower risk of several common ...
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 ...
Mayer-Rokitansky-Küster-Hauser (MRKH) syndrome is a rare condition that impacts the female reproductive system. Its characteristic feature is an underdeveloped or absent uterus and vagina, but ...
Long QT syndrome is a leading cause of sudden death in young persons, with a prevalence exceeding 1 in 2000. It is characterized by prolongation of the QT interval, aberrant T-wave morphologic ...
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 ...
Sanfilippo syndrome, also called mucopolysaccharidosis (MPS) type III, is a genetically inherited metabolic disorder that causes nervous system issues and other symptoms in children. This syndrome is ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results