«
»

Top

Latest articles published - Acatalasemia

US Approves First RNA Interference Drug

3-methylcrotonyl-CoA carboxylase deficiency
Acatalasemia
Aceruloplasminemia
Aciduria
Addison's disease
Adenine phosphoribosyltransferase deficiency
Adrenal insufficiency
Alkaptonuria
Alpha 1-antitrypsin deficiency
Alpha-mannosidosis
Amyloidosis
Androgen insensitivity syndrome
Athyreosis
Bariatric surgery
Bartter syndrome
Berardinelli-Seip congenital lipodystrophy
Biermer's disease
Biotinidase deficiency
Branching enzyme deficiency
Carbamoylphosphate synthetase deficiency
Carnitine palmitoyl transferase II deficiency
Catecholamine-secreting tumor
Chronic hepatic porphyria
Citrullinemia
Colchicine poisoning
Congenital adrenal hyperplasia
Congenital disorder of glycosylation
Congenital hypogonadotropic hypogonadism
Congenital isolated thyroxine-binding globulin deficiency
Congenital sucrase-isomaltase deficiency
Craniopharyngioma
Crigler-Najjar syndrome
Cushing syndrome
Cystathioninuria
Cystinosis
Cystinuria
Diabetes insipidus
Erythropoietic protoporphyria
Familial amyloid polyneuropathy
Fluid retention
Fructose-1
6-bisphosphatase deficiency
Galactosemia
Gaucher disease
Gitelman syndrome
Glutaryl-CoA dehydrogenase deficiency
Glycogen storage disease type 2
Gout disease
Hartnup syndrome
Histidinemia
Homocystinuria due to cystathionine beta-synthase deficiency
Hurler syndrome
Hurler-Scheie syndrome
Hyperargininemia
Hyperlipoproteinemia
Hyperlipoproteinemia type 3
Hyperoxaluria
Hyperparathyroidism
Iminoglycinuria
Isolated congenital hyperinsulinism
Isovaleric acidemia
Krabbe disease
Lemierre syndrome
Long chain 3-hydroxyacyl-CoA dehydrogenase deficiency
Maple syrup urine disease
Maternally-inherited diabetes and deafness
Medium chain acyl-CoA dehydrogenase deficiency
Metabolic syndrome
Metachromatic leukodystrophy
Methylmalonic acidemia
Mitochondrial cytopathies
Mucolipidosis type 2
Mucopolysaccharidosis
Neonatal diabetes mellitus
Nephrogenic diabetes insipidus
Niemann-Pick disease
Non-diabetic hypoglycemia
Non-ketotic hyperglycinemia
Nonacquired combined pituitary hormone deficiency
Nutrition / Metabolism / Endocrine glands
Obesity
Oculocerebrorenal syndrome
Ornithine transcarbamylase deficiency
Pelizaeus-Merzbacher disease
Phenylketonuria
Pickwick syndrome
Pituitary adenoma
Pituitary insufficiency
Porphyria
Porphyria cutanea tarda
Progeria
Propionic acidemia
Sandhoff disease
Sarcosinemia
Saturnism
Scheie syndrome
Scurvy
Secondary Adrenal Insufficiency
Severe combined immunodeficiency due to adenosine deaminase deficiency
Sialidosis type 1
Sialidosis type 2
Sjögren-Larsson syndrome
Stiff person syndrome
Tay-Sachs disease
Triple H syndrome
Tyrosinemia type 1
Vitamin B12 deficiency
Vitamin D deficiency
Wilson disease
Wolfram syndrome
Wolman disease
Zollinger-Ellison syndrome

US Approves First RNA Interference Drug

Alnylam’s Onpattro (patisiran) can treat a rare hereditary disease by silencing a specific gene. The first therapy based on RNA interference (RNAi) gene silencing has been approved by the US Food and Drug Administration (FDA). Alnylam...

Read the article