Data from the first patients enrolled into Regenxbio's trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the ...

MPS disorders are inherited in an autosomal recessive manner, except for MPS II (Hunter syndrome), which is X-linked. Symptoms vary depending on the specific type ... like MPS I (Hurler syndrome ...

or MPS II, and RGX-111 for Hurler syndrome, or MPS I. Under the deal, REGENXBIO (NASDAQ:RGNX) will receive $110M upfront and up to $700M in potential milestone payments, plus double-digit ...