Seelos Therapeutics‘ investigational new drug application for SLS-005 (trehalose) has been accepted by the U.S. Food and Drug Administration…
Articles by José Lopes, PhD
The youngest Sanfilippo syndrome type A children treated with the gene therapy candidate ABO-102 continue to have normal neurocognitive development…
Patients with Sanfilippo syndrome type C may develop an accumulation of heparan sulfate in specialized cells involved in the…
A Phase 2/3 trial of the Sanfilippo syndrome type A gene therapy candidate LYS-SAF302 has dosed its first patient…
Hernias, flat feet, joint rigidity, and keen misalignment are among the clinical symptoms at diagnosis in patients with mucopolysaccharoidosis (MPS) including…
Patients with Sanfilippo syndrome type A function below a two-year-old level in terms of communication, daily living skills and socialization,…
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…
Sanfilippo syndrome type A patients treated with the gene therapy candidate ABO-102 continue to show robust and sustained…
Treating Sanfilippo syndrome type A (SSA) may be possible with a fusion protein that effectively reaches the brain, according…
The first fly model of Sanfilippo syndrome type A has been developed and characterized, providing new data on genes…