viagra in chennai toyota tacoma rock crawler viagra mit ec karte kaufen erectile dysfunction medication

Genetic & Metabolic

This unit focus on providing meaningful advances to patients who live with serious and life-threating rare genetic diseases, operates through 6 main areas:
  • Metabolic diseases
  • immunology/inflammation
  • Oncology
  • Cardiology neonatology
  • Hepatology
  • hemophilia
  • Brineura (cerliponase alfa) for CLN2 disease.
  • Vimizim (elosulfase alfa) for Morquio A Syndrome MPS IVA.
  • Kuvan (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution for PKU.
  • Naglazyme (galsulfase) for MPS VI.
  • Firdapse (amifampridine phosphate) (currently approved in the EU only) for LEMS.
  • Ammonul® (sodium phenylacetate and sodium benzoate treatment for acutehyperammonemia.
  • Ammonaps® (sodium phenylbutyrate) treatment for chronic hyperammonemia.
  • Ravicti (Glycerol phenylacetate) treatment for Chronic hyperammonemia.
  • Orfadin (nitisinone) treatment for tyrosinemia type 1.
  • Kineret® (anakinra) treatment for Neonatal onset multisystem inflammatory disease.
  • Alprolix Eftrenonacog alfa treatment for hemophilia B.
  • Elocta® efmoroctocog alfa treatment for hemophilia A.
  • Aldurazyme(laronidase) treatment for MPS type I.
  • Myozyme(alglucosidase alfa) treatment for pompe disease.
  • Cerezyme(imiglucerase) treatment for Gaucher disease.
  • Cerdelga (eliglustat) treatment for adult Gaucher disease.
  • Carbaglu (carglumic acid) treatment of acute hyperammonemia.
  • Cosmegen (Dactinomycin) treatment regimen for metastatic, nonseminomatous testicular cancer.
  • Cystadane (betaine anhydrous for oral solution) treatment of homocystinuria.
  • Cystadrops (Cysteamin) treatment of corneal cystine crystal deposits.
  • Cystagon (Cysteamine bitartrate) treatment of nephropathic cystinosis.
  • Normosang (human hemin) treatment of acute attacks of hepatic porphyria.
  • Pedea (ibuprofen)Treatment of a haemodynamically significant patent ductus arteriosus.
  • Vedrop (tocofersolan) vitamin E deficiency.
  • Wilzin (zinc acetate dehydrate) Treatment of Wilson's disease.