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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 goal of this clinical trial is to test cannabidiol in Sanfilippo syndrome. The main questions it aims to answer are: 1) determine the safety of cannabidiol…
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.…
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…
Background: Ollier disease (OD) and Maffucci syndrome (MS) are rare disorders that increase the risk of cancers in cartilage tissue. These tumors can lead to s…
The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersens…
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
MOSAIC aims to determine whether oro-dental morphological anomalies, particularly palatal morphology, associated with rare bone and cartilage diseases can be pr…
This is a multiregional open-label extension (OLE) to assess the safety, tolerability, and efficacy of long-term treatment with tividenofusp alfa (DNL310), an i…