The U.S. Food and Drug Administration (FDA) has accepted an investigational new drug (IND) application for Swedish Orphan Biovitrum AB‘s (Sobi) enzyme replacement therapy SOBI003, for the treatment of Sanfilippo syndrome type A. The FDA also granted the therapy fast track status, which is expected to accelerate its clinical development and regulatory…
News
Mild to moderate hearing loss is common among children and young adults with different mucopolysaccharidosis (MPS) diseases, including Sanfilippo syndrome, a small study reports. The study, “Audiometric evaluation in individuals with muco-polysaccharidosis,” appeared in the journal Clinics. MPS disorders are identified by deficiencies in lysosomal enzymes involved…
Heparan sulfate accumulation is not a good indicator of Sanfilippo syndrome type A symptom onset, a new mouse model of the disease suggests. This finding, reported in the study, “A novel conditional Sgsh knockout mouse model recapitulates phenotypic and neuropathic deficits of Sanfilippo syndrome,” reiterates other…
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