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DMD

A...

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Farber Disease Natural History Study

Brief Summary: The primary objective of this study is to establish the natural history of Farber disease (acid ceramidase deficiency) through the collection and analysis of retrospective and prospective data...

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Phase IIb trial in adult MPS VI ( Mucopolysacridosis )

Date of Publication: 2.8.2018 Name of the diseases : Phase IIb trial in adult MPS VI ( Mucopolysacridosis ) patients Name of the Drug:  Odiparcil  (   Odiparcil is a small molecule and...

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Clinical trial on – Gaucher disease (GD)

Place : Jaslok Hospital and Research Centre, Mumbai Study – This study is being conducted for the drug “Orphazyme A/S” Eligibility : The study population required for the study is...

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