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Treating Rare Pediatric Neurological Diseases with Enzyme Replacement Therapy

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We believe that intracerebroventricular enzyme replacement therapy holds exciting potential to treat rare pediatric neurological diseases. We’ve begun with the development of tralesinidase alfa (AX 250) to treat Sanfilippo syndrome type B.


By delivering therapy directly to the cells of the central nervous system, enzyme replacement therapies hold promise to slow progression of this and other lysosomal storage diseases that cause neurological symptoms, thereby offering new hope to children living with these disorders.

Allievex Drug Development Pipeline

Sanfilippo Syndrome Type B (AX 250)

Children with Sanfilippo syndrome type B develop normally for their first year or two. Due to a missing enzyme, however, toxins accumulate in the brain and spinal cord, causing developmental delays, seizures, mobility difficulties, and progressive neurological disability. About 1 in 150,000 newborns have Sanfilippo syndrome type B.

Sanfilippo Syndrome Type A (AX 313)

Sanfilippo syndrome type A (MPS IIIA) is another lysosomal storage disease with symptoms similar to Type B, although they may occur at a younger age and with increased severity. More children have Sanfilippo syndrome type A than type B, with an approximate incidence rate of 1 in 100,000 newborns. There are no approved therapies for Sanfilippo syndrome type A.

GM2 Gangliosidosis (AX 451)

GM2 Gangliosidosis, also known as Tay-Sachs disease, is a lysosomal storage disorder that progressively destroys nerve cells in the brain and spinal cord. Onset typically occurs in infancy, but it can also begin during childhood or even early adulthood. Symptoms include developmental delay, seizures, loss of hearing, and cognitive impairment. Currently, there are no approved therapies for this disease. The incidence of GM2 Gangliosidosis is 1 in 222,000 newborns.

GM1 Gangliosidosis (AX 552)

GM1 Gangliosidosis is another lysosomal storage disorder that progressively destroys nerve cells in the brain and spinal cord. Similar to GM2 Gangliosidosis, onset can begin anywhere from infancy to adulthood, and the disease causes progressive loss of intellectual and physical function. The disease may cause cognitive impairment, liver and spleen enlargement, speech difficulties, and seizures. No effective treatment exists to slow or reverse the progression of GM1 Gangliosidosis. The incidence of GM1 is 1 in 100,000 to 200,000 newborns.

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