
We focus Rare disordes
Neurology disorders
LNS
TSC
LHON
Blood Disorders
PNH
MDS
kidney Rare
C3
FSGS
MN
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Paroxysmal nocturnal hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic,
progressive, debilitating and life-threatening ultra-rare
blood disorder characterized by complement-mediated
hemolysis (destruction of red blood cells).
Lesch-Nyhan syndrome
Lesch Nyhan syndrome is a condition characterized by neurological and behavioral abnormalities and the overproduction of uric acid in the body. It occurs almost exclusively in males.
C3 glomerulopathy
C3G stands for complement 3 glomerulopathy. The “C3” refers to a blood protein that plays a key role in normal immunity. The “G” is for glomerulopathy, meaning damage to the glomeruli in the kidney. Glomeruli filter your blood to get rid of toxins and make urine.