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Mucopolysaccharidosis Disorders
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis Disorders trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis Disorders trials may be worth asking aboutNorth America
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The recovery of transit after surgery is an important parameter in postoperative evaluation. It generally reflects simple postoperative outcomes and allows the…
Background: There are 461 conditions that affect the bones (skeletal disorders). Many of these are not well understood and do not have any specific treatments.…
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
This is a long-term, multicenter, non-interventional study of children ages 2.5 to \<17 years with hypochondroplasia (HCH).
This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \< 3 years old with ACH…
Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their prim…
The primary objectives of this study are to evaluate the effects of Tralesinidase Alfa (TA) on cognition