Hunter syndrome
Regenxbio announces positive data from trial for MPS II treatment
February 8, 2024Research 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 …
Shire lifted by rare disease approvals
January 26, 2015Sales 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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