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Genes Treatments
Naglazyme: Used for Sanfilippo syndrome type A.
Brief information about it: Naglazyme is an enzyme replacement therapy (ERT) used to treat Sanfilippo syndrome type A, a rare…
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Genes Treatments
Aldurazyme: Used for Hunter syndrome.
Brief information about it: Aldurazyme is an enzyme replacement therapy (ERT) used to treat Hunter syndrome, a rare genetic disorder…
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Genes Treatments
Lumizyme: Used for Fabry disease.
Brief information about it: Lumizyme is an enzyme replacement therapy (ERT) used to treat Fabry disease, a rare genetic disorder…
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Genes Treatments
Myozyme: Used for Pompe disease.
Brief information about it: Myozyme is an enzyme replacement therapy (ERT) used to treat Pompe disease, a rare genetic disorder…
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Genes Treatments
Ceredase: Used for Gaucher disease.
Brief information about it: Ceredase is an enzyme replacement therapy (ERT) used to treat Gaucher disease, a rare genetic disorder…
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Genes Treatments
Spark-101: Being developed for Leber congenital amaurosis (LCA) type 10.
Brief information about it: Spark-101, also known as voretigene nepavehvec-rzyl, is a gene therapy being developed for the treatment of…
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Genes Treatments
OTC-601: Being developed for Huntington’s disease.
Brief information about it: OTC-601 is an investigational therapy being developed for the treatment of Huntington’s disease, a neurodegenerative disorder…
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Genes Treatments
RGX-314: Being developed for Duchenne muscular dystrophy.
Brief information about it: RGX-314 is a gene therapy being developed for the treatment of Duchenne muscular dystrophy (DMD), a…
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Genes Treatments
CORTEX-1: Being developed for cystic fibrosis.
Brief information about it: CORTEX-1 is a gene therapy being developed for the treatment of cystic fibrosis (CF), a genetic…
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Genes Treatments
AVXS-101: Being developed for spinal muscular atrophy (SMA).
Brief information about it: AVXS-101, now known as Spinraza, is a gene therapy medication used to treat spinal muscular atrophy…
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