Hunter syndrome

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Regenxbio announces positive data from trial for MPS II treatment

February 8, 2024
Research and Development Hunter syndrome, MPS II, Neurology, Regenxbio, clinical trial

Regenxbio has announced topline results from the phase 1/2/3 CAMPSIITE trial of RGX-121 for the treatment of patients up to …

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Shire lifted by rare disease approvals

January 26, 2015
Sales and Marketing HGT-2310, Hunter syndrome, SHP609, Shire, fast track, idursulfase-IT, rare disease

Shire’s investigational drug for a rare neurological disease has been accepted into the FDA’s fast-track approval process. The drug, SHP609, …

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