José Lopes, PhD, managing science editor —

Seelos Therapeutics‘ investigational new drug application for SLS-005 (trehalose) has been accepted by the U.S. Food and Drug Administration (FDA) for the treatment of Sanfilippo syndrome. The company can now start a Phase 2b/3 trial to test its investigational therapy in patients with Sanfilippo syndrome types A…

The youngest Sanfilippo syndrome type A children treated with the gene therapy candidate ABO-102 continue to have normal neurocognitive development up to 18 months after treatment in an ongoing Phase 1/2 trial, the therapy’s developer, Abeona Therapeutics, has announced. Early results also show favorable safety up to two years…

Patients with Sanfilippo syndrome type C may develop an accumulation of heparan sulfate in specialized cells involved in the filtering of molecules within the kidney, among other alterations, a mouse study finds. The study, “HGSNAT Enzyme Deficiency Results in Accumulation of Heparan Sulfate in Podocytes and…

A Phase 2/3 trial of the Sanfilippo syndrome type A gene therapy candidate LYS-SAF302 has dosed its first patient in Europe. Called AAVance, the open-label trial (NCT03612869) is assessing the safety and efficacy of a one-time delivery of LYS-SAF302. The team intends to recruit a total of 20…

Hernias, flat feet, joint rigidity, and keen misalignment are among the clinical symptoms at diagnosis in patients with mucopolysaccharoidosis (MPS) including Sanfilippo syndrome, according to Mexican study. The research, “Diagnosis of Mucopolysaccharidosis Based on History and Clinical Features: Evidence from the Bajio Region of Mexico,” was published in…

Patients with Sanfilippo syndrome type A function below a two-year-old level in terms of communication, daily living skills and socialization, according to a small follow-up study of the later stages of the disorder. Motor skills were slightly more preserved. The study also revealed that parent burden shifts from behavioral control…

AGT-184 has been granted orphan drug designation for the treatment of Sanfilippo syndrome type A by the U.S. Food and Drug Aministration. Sanfilippo syndrome type A is caused by mutations in the gene that provides instructions to make the enzyme N-sulfoglucosamine sulfohydrolase (SGSH). This leads to the accumulation of a complex sugar molecule…

Sanfilippo syndrome type A patients treated with the gene therapy candidate ABO-102 continue to show robust and sustained clinical improvements in a Phase 1/2 trial, its developer, Abeona Therapeutics, reports. The company presented updated results of its open-label study (NCT02716246) at the recent American Society for…

Treating Sanfilippo syndrome type A (SSA) may be possible with a fusion protein that effectively reaches the brain, according to lab research with mice. The study, “Reduction in Brain Heparan Sulfate with Systemic Administration of an IgG Trojan Horse-Sulfamindase Fusion Protein in the Mucopolysaccharidosis Type IIIA Mouse,” appeared…

The first fly model of Sanfilippo syndrome type A has been developed and characterized, providing new data on genes involved in disease development. The study, “Neuronal-specific impairment of heparan sulfate degradation in Drosophila reveals pathogenic mechanisms for Mucopolysaccharidosis type IIIA,” appeared in the journal Experimental Neurology. Sanfilippo…