<unk> , marketed as Brineura, is an enzyme replacement treatment for Batten disease, a neurodegenerative lysosomal storage disease. Specifically,<unk>  is meant to slow loss of motor function in symptomatic children over three years old with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). The disease is also known as tripeptidyl peptidase-1 (TPP1) deficiency, a soluble lysosomal enzyme deficiency. Approved by the United States Food and Drug Administration (FDA) on 27 April 2017, this is the first treatment for a neuronal ceroid lipofuscinosis of its kind, acting to slow disease progression rather than palliatively treat symptoms by giving patients the TPP1 enzyme they are lacking. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.
Cerliponase alfa
Avalglucosidase alfa
Alglucosidase alfa
Sebelipase alfa
Velmanase alfa
Vestronidase alfa
Imiglucerase
Sulfatide