Gene therapy is a promising approach for a number of rare genetic diseases as it has the potential to treat the underlying cause. Here is more information about gene therapy, its challenges, and its current status as a treatment for Sanfilippo syndrome. About gene therapy Gene therapy is a treatment…
SOBI003, Swedish Orphan Biovitrum AB (Sobi)’s chemically modified version of sulfamidase — the missing enzyme in Sanfilippo syndrome type A — is superior to its original version at reaching and persisting in the brain, a study in rats suggests. These findings further support the already ongoing…
Several approaches are currently being investigated to allow the delivery of novel therapeutic agents into the central nervous system and target neurological symptoms in patients with mucopolysaccharidosis disorders, including Sanfilippo syndrome. Although patients with mucopolysaccharidosis (MPS) disorders can suffer from neurological disorders that include neurocognitive decline and behavioral problems, treating these…
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