Sanfilippo Syndrome

• CLINICAL_TRIALS research · 2026-04-06
  Clinical Trial Update: Study of Cannabidiol in Sanfilippo Syndrome [Phase 2, Phase 3]

  A cannabidiol study for Sanfilippo syndrome (Mucopolysaccharidosis type III) is recruiting participants for Phase 2 and Phase 3 at the Lundq…

• CLINICAL_TRIALS research · 2025-08-20
  Clinical Trial Update: An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III [Phase 2, Phase 3]

  An open-label dose-escalation study (Phase 2/Phase 3) is evaluating the safety, tolerability, and pharmacologic properties of high-dose ambr…

• CLINICAL_TRIALS trial_result · 2023-07-25
  Clinical Trial Update: Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease [Phase 1, Phase 2]

  A phase 1/2 clinical trial of ABO-102 for MPS IIIA (Sanfilippo syndrome) was terminated; results have been posted.

• CLINICAL_TRIALS trial_result · 2020-04-20
  Clinical Trial Update: An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. [Phase 2]

  An extension study to assess safety and efficacy in pediatric MPS IIIA patients who participated in a prior study (Phase 2; terminated).

• CLINICAL_TRIALS trial_result · 2017-03-03
  Clinical Trial Update: Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease [Phase 2]

  Phase 2 randomized trial assessing safety and efficacy of rhHNS administered via IDDD in pediatric Sanfilippo syndrome (MPS IIIA); study com…

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