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Dx Lysosomal Storage Disease Treatments: Read more...


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Observation:
Lysosomal Storage Disease

N.H.S. Choices
states:
“Lysosomal Storage Disorders:
Lysosomal Storage Disorders (LSDs) are a group of rare genetic storage disorders, characterised by specific lysosomal enzyme deficiencies. Some Lysosomal Storage Disorders can be treated using enzyme replacement therapies (ERTs).

There are licensed ERTs for six LSDs:
*Gaucher’s disease
*Anderson-Fabry’s disease
*Mucopolysaccharidosis type I (MPSI, which occurs as Hurler’s syndrome, Hurler-Scheie syndrome and Scheie syndrome)
*Mucopolysaccharidosis type VI (MPSVI or Maroteaux Lamy syndrome)
*Pompe’s disease
*Mucopolysaccharidosis type II (MPSII)

Medications Used in Treatment:
1. Enzyme Replacement Therapies: Lumizyme® Myozyme®/alglucosidase alfa, Cereyme®/imiglucerase. Naglazyme®/galsulfase, Aldurazyme®/laronidase, Elaprases®/idursulfase, Vpriv®/velaglucerase alfa, Elelyso®/taliglycerase alfa, Fabrazyme®/agalsidase beta
2. Cystaran® Cystagon® Procysbi®/ cysteamine
3. GSC Inhibitors: Zavesca®/miglustat
4. Lysosomal Disease: Enzyme Replacement Therapy: Xuriden®

Suggested Links
Organizations Addressing Lysosomal Storage Diseases


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